Children's Hospital Medical Center

United States of America

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C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues 72
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids 56
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1.

COMPOSITIONS AND METHODS FOR TREATING ALLERGIC INFLAMMATORY CONDITIONS

      
Application Number 18971180
Status Pending
Filing Date 2024-12-06
First Publication Date 2025-03-27
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Azouz, Nurit P.
  • Rothenberg, Marc E.

Abstract

The invention provides methods for increasing SPINK7 activity in a target tissue of a subject by inhibiting urokinase plasminogen activator (uPA) proteolytic activity in the target tissue, and related methods and compositions.

IPC Classes  ?

  • A61K 31/37 - Coumarins, e.g. psoralen
  • A61K 31/352 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. cannabinols, methantheline
  • A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
  • A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
  • A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
  • A61K 38/00 - Medicinal preparations containing peptides
  • A61K 38/55 - Protease inhibitors
  • A61K 38/57 - Protease inhibitors from animalsProtease inhibitors from humans
  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • A61P 37/08 - Antiallergic agents
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

2.

SYSTEMS AND METHODS FOR MITIGATING DIVERSITY, EQUITY AND INCLUSION BIAS IN CLINICAL ARTIFICIAL INTELLIGENCE MODELS

      
Application Number US2024047471
Publication Number 2025/064663
Status In Force
Filing Date 2024-09-19
Publication Date 2025-03-27
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Pestian, John
  • Glauser, Tracy
  • Ive, Julia
  • Santel, Daniel

Abstract

A framework is provided to assess and mitigate bias in clinical artificial intelligence and machine learning. The framework includes features that demonstrate usefulness in indicating the potential causes of bias and ways to mitigate them. The framework measures classification parity, as well as the effect of biased words on predictions. Techniques are provided that reduce bias in training data (derived from electronic health records) by filtering the salient clinical information and neutralizing biased words. In an embodiment, training data for a machine learning engine is rewritten to mitigate bias in the training dataset, where the re-writing step includes a step of information density filtering on electronic health record training data and/or replacing biased words in electronic health record training data with relevant neutral versions of the biased words.

IPC Classes  ?

  • G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
  • G16H 50/70 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for mining of medical data, e.g. analysing previous cases of other patients
  • G16H 20/10 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients
  • G16H 20/70 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to mental therapies, e.g. psychological therapy or autogenous training

3.

METHODS FOR THE TREATMENT OF LUPUS NEPHRITIS

      
Application Number US2024046306
Publication Number 2025/059250
Status In Force
Filing Date 2024-09-12
Publication Date 2025-03-20
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Brunner, Hermine, I.
  • Devarajan, Prasad

Abstract

Disclosed are methods for non-invasively classifying an individual diagnosed with lupus nephritis (LN) as a responder, a partial responder or a non-responder to lupus nephritis induction therapy. The method generally comprises (a) comparing a first Renal Activity Index for Lupus (RAIL) score and a second RAIL score, based on the detection of neutrophil gelatinase-associated lipocalin (NGAL), kidney injury molcculc-1 (KIM-1), monocyte chemotactic protein 1 (MCP-1), adiponectin, hemopexin and ceruloplasmin in a urine sample from the individual. Based on the RAIL score, the individual can be classified as a responder, a partial responder or a complete responder, and therapy can be appropriately adjusted.

IPC Classes  ?

  • G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
  • G01N 33/564 - ImmunoassayBiospecific binding assayMaterials therefor for pre-existing immune complex or autoimmune disease
  • G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment

4.

METHODS OF TREATING EOSINOPHILIC COLITIS

      
Application Number 18728969
Status Pending
Filing Date 2023-01-17
First Publication Date 2025-03-13
Owner Children's Hospital Medical Center (USA)
Inventor
  • Rothenberg, Marc E.
  • Shoda, Tetsuo

Abstract

Disclosed are methods for treating active eosinophilic colitis (EoC), or in certain aspects, inflammatory bowel disease (IBD), in an individual in need thereof. In one aspect, the methods may comprise a) assaying a tissue sample obtained from a colon of an individual who may be in need of such treatment, wherein the assaying comprises detecting expression of one or more gene of a transcriptome gene set; b) calculating a score based on the expression of one or more gene of a transcriptome gene set; and c) selecting a tissue sample that exhibits a score indicative of EoC or IBD. The methods may further comprise administering an EoC or IBD therapy to the individual whose tissue sample exhibited a score indicative of having EoC or IBD.

IPC Classes  ?

  • C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
  • C12Q 1/6851 - Quantitative amplification
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

5.

MPOX VACCINE COMPOSITIONS AND METHODS OF USING SAME

      
Application Number US2024044023
Publication Number 2025/049467
Status In Force
Filing Date 2024-08-27
Publication Date 2025-03-06
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor Tan, Ming

Abstract

Disclosed are vaccine compositions, in particular, polyvalent icosahedral compositions for presentation of an mpox antigen. The disclosed compositions may contain an S particle comprising a norovirus (NoV) S domain protein and an mpox antigen, which may be linked via a linker protein domain operatively connected to the norovirus S domain protein and an mpox antigen. Fusion proteins for producing the vaccine compositions, and methods of using the disclosed vaccine composition are also provided.

IPC Classes  ?

6.

IDENTIFICATION OF TARGETS FOR IMMUNOTHERAPY IN MELANOMA USING SPLICING-DERIVED NEOANTIGENS

      
Application Number US2024044819
Publication Number 2025/050009
Status In Force
Filing Date 2024-08-30
Publication Date 2025-03-06
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Salomonis, Nathan
  • Li, Guangyuan

Abstract

Immunogenic targets associated with one or more types of cancer, wherein the immunogenic target includes a shared splicing amino acid-based neoantigen present in any of SEQ ID NOs: 1-2545, or a nucleotide coding therefor, are described, as well as uses thereof, and methods of identifying such immunogenic targets. Particular embodiments relate to immunotherapies for treating a type of cancer, and methods of treating a type of cancer by administering such immunotherapies, wherein the immunotherapy includes an immunogenic target including a shared splicing amino acid-based neoantigen present in any of SEQ ID NOs: 1-2545, or a nucleotide coding therefor.

7.

COMPOSITIONS AND METHODS FOR TREATING TRANSPLANT ASSOCIATED THROMBOTIC MICROANGIOPATHY IN BLEEDING PATIENTS

      
Document Number 03242993
Status Pending
Filing Date 2022-12-21
Open to Public Date 2025-02-27
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Jodele, Sonata
  • Mizuno, Kana
  • Vinks, Alexander A.

Abstract

The instant disclosure relates to methods for the treatment of an individual having TA-TMA, In one aspect, the methods encompass administration of a C5 inhibitor, more particularly eculizumab. or an antigen-binding fragment thereof. The disclosed methods, in one aspect, may be used for the treatment of individuals having TA-TMA and clinically significant bleeding.

IPC Classes  ?

  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid
  • A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
  • C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans

8.

COMPOSITIONS AND METHODS FOR TREATING TRANSPLANT ASSOCIATED THROMBOTIC MICROANGIOPATHY IN BLEEDING PATIENTS

      
Application Number 18722118
Status Pending
Filing Date 2022-12-21
First Publication Date 2025-02-27
Owner Children's Hospital Medical Center (USA)
Inventor
  • Jodele, Sonata
  • Mizuno, Kana
  • Vinks, Alexander A.

Abstract

The instant disclosure relates to methods for the treatment of an individual having TA-TMA, In one aspect, the methods encompass administration of a C5 inhibitor, more particularly eculizumab, or an antigen-binding fragment thereof. The disclosed methods, in one aspect, may be used for the treatment of individuals having TA-TMA and clinically significant bleeding.

IPC Classes  ?

  • C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61P 7/02 - Antithrombotic agentsAnticoagulantsPlatelet aggregation inhibitors
  • A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
  • G16H 20/17 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients delivered via infusion or injection

9.

AVAIS

      
Serial Number 99059803
Status Pending
Filing Date 2025-02-27
Owner Children's Hospital Medical Center ()
NICE Classes  ? 44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services

Goods & Services

Pediatric and adolescent surgical services, namely, cardiothoracic surgery; cardiothoracic services; diagnosis and treatment of cardiothoracic ailments; medical treatment in the field of cardiothoracic surgery; medical procedures for cardiothoracic purposes

10.

MULTI-CYCLIC IRAK1 AND IRAK4 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number US2024040403
Publication Number 2025/029929
Status In Force
Filing Date 2024-07-31
Publication Date 2025-02-06
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES (USA)
  • KUROME THERAPEUTICS, INC. (USA)
Inventor
  • Hoyt, Scott, Bryan
  • Starczynowski, Daniel, T.
  • Thomas, Craig, Joseph
  • Rosenbaum, Jan, Susan
  • Bennett, Joshua
  • Hayes, Gregory, M.

Abstract

The present disclosure provides a method of treating an inflammatory disease or disorder, acute myeloid leukemia (AML), or myelodysplastic syndrome (MDS) in a subject, the method comprising administering to the subject a compound that inhibits IRAK1 and IRAK4. The present disclosure further provides a method of determining a subject with a disease/disorder that can be treated by the administration of a compound which inhibits IRAK1 and IRAK4.

IPC Classes  ?

  • A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
  • A61K 31/44 - Non-condensed pyridinesHydrogenated derivatives thereof
  • A61K 31/445 - Non-condensed piperidines, e.g. piperocaine

11.

SELF-INACTIVATING LENTIVIRAL VECTOR ENCODING BETA- OR GAMMA-GLOBIN

      
Application Number 18737201
Status Pending
Filing Date 2024-06-07
First Publication Date 2025-01-30
Owner Children's Hospital Medical Center (USA)
Inventor Malik, Punam

Abstract

Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increase a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention further relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia, a lysosomal storage disease. The invention further relates to a method of improving and/or correcting one or more central nervous system (CNS) abnormalities caused by one or more lysosomal storage disease. The invention further relates to methods of improving titer in transfection-based bioreactor culture production or transfection-based production systems using eukaryotic cells.

IPC Classes  ?

  • C12N 15/86 - Viral vectors
  • C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
  • C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells

12.

HEART VALVE REPLACEMENT

      
Application Number AU2024050744
Publication Number 2025/010475
Status In Force
Filing Date 2024-07-11
Publication Date 2025-01-16
Owner
  • THE UNIVERSITY OF SYDNEY (Australia)
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Naficy, Sina
  • Lee, Aeryne
  • Fletcher, David Frederick
  • Winlaw, David Scott
  • Dehghani, Fariba

Abstract

A method (100) of fabricating a heart valve replacement, the method (100) comprising: obtaining a representation (200) of a native heart valve (202) of a subject; obtaining one or more profiles of the native heart valve (202) from the representation (200) of the native heart valve (202), the one or more profiles defining one or more geometries of the native heart valve (202); deriving one or more mathematical functions from the one or more profiles; generating a model (400) of the heart valve replacement using the one or more mathematical functions; and fabricating the heart valve replacement based on the model (400) of the heart valve replacement.

IPC Classes  ?

13.

JOINT PROFILING OF GENETIC VARIANTS, DNA METHYLATION, GPC METHYLTRANSFERASE FOOTPRINTS, 3D GENOME AND TRANSCRIPTOME

      
Application Number 18712405
Status Pending
Filing Date 2022-11-22
First Publication Date 2025-01-09
Owner Children's Hospital Medical Center (USA)
Inventor
  • Wang, Li
  • Liu, Yaping

Abstract

The present invention provides a system and methods for detecting long-range cis-regulatory element (CRE) activities in a nucleic acid molecule obtained from a cell sample, the system comprising: one or more components for measuring multi-omics from a single nucleic acid molecule obtained from the cell sample, wherein the multi-omics measurements comprise one or more of: three-dimensional chromosomal conformation, CpG methylation, GpC accessibility, single nucleotide polymorphisms (SNPs); or one or more combinations thereof, and one or more components for measuring a transcriptome of the cell sample; wherein the system is capable of profiling a plurality of long-range CREs within the nucleic acid molecule obtained from the cell sample.

IPC Classes  ?

  • G16B 20/20 - Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
  • C12Q 1/48 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving transferase
  • C12Q 1/6806 - Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
  • C12Q 1/6827 - Hybridisation assays for detection of mutation or polymorphism
  • C12Q 1/6869 - Methods for sequencing

14.

VASCULARIZED BRAIN ORGANOIDS AND METHODS OF MAKING AND USING THE SAME

      
Application Number US2024035718
Publication Number 2025/006679
Status In Force
Filing Date 2024-06-27
Publication Date 2025-01-02
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Guo, Ziyuan
  • Dao, Lan

Abstract

Disclosed herein are organoid models of the blood-brain barrier, optionally having a disease associated feature, such as a cerebral cavernous malformation (CCM)-like feature, compositions comprising the same, methods for making the same from pluripotent stem cells, and methods of using the same. These organoids exhibit the complex organization of cells including neurons, endothelial cells, glial cells, and pericytes resembling the natural blood-brain barrier structure. The organoids may also exhibit disease like characteristics, such as having a cerebral cavernous malformation (CCM)-like features. These organoids may be used for studying the functions of the blood-brain barrier and cerebrovascular disorders.

IPC Classes  ?

  • C12N 5/00 - Undifferentiated human, animal or plant cells, e.g. cell linesTissuesCultivation or maintenance thereofCulture media therefor

15.

METHODS OF MATRIX-FREE SUSPENSION CULTURE

      
Application Number US2024035082
Publication Number 2024/263961
Status In Force
Filing Date 2024-06-21
Publication Date 2024-12-26
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Kasendra, Magdalena
  • Boylin, Katherine

Abstract

Disclosed herein are methods of utilizing and forming suspension cultures of pluripotent stem cells (PSC), and differentiated cells, spheroids and organoids derived from PSCs (e.g., at industrial efficiency and/or scalability), and compositions comprising the same. These methods may be performed in suspension culture, without the use of basement membrane matrices, during PSC maintenance and expansion, as well as during differentiation of PSCs into differentiated cells and organoids, for example definitive endoderm (DE), hindgut spheroids (HGS), and intestinal organoids (IO). In some aspects, the methods may be xeno-free, and may be performed as per Good Manufacturing Practices (GMP). Also disclosed herein are methods controlling the polarity of epithelial cells in IOs, wherein the apical layer is oriented to the outside of the organoid, or alternatively to the inside of the organoid. Also disclosed are uses of the methods and compositions for transplantation and treatment.

IPC Classes  ?

  • C12N 5/00 - Undifferentiated human, animal or plant cells, e.g. cell linesTissuesCultivation or maintenance thereofCulture media therefor
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

16.

COMPOSITIONS AND METHODS FOR FORECASTING RESPONSE TO LUPUS NEPHRITIS (LN)THERAPY

      
Application Number 18602128
Status Pending
Filing Date 2024-03-12
First Publication Date 2024-12-19
Owner Children's Hospital Medical Center (USA)
Inventor
  • Brunner, Hermine I.
  • Bennett, Michael R.
  • Devarajan, Prasad

Abstract

Disclosed herein are methods for forecasting response to lupus nephritis (LN) therapy in individual diagnosed with childhood-onset SLE (cSLE). The methods may include the step of detecting each protein in a protein set in a sample obtained from an individual in need thereof. The protein set may include ceruloplasmin, kidney injury molecule 1 (KIM-1), monocyte chemotactic protein 1 (MCP-1), adiponectin, hemopexin, and NGAL.

IPC Classes  ?

  • G01N 33/564 - ImmunoassayBiospecific binding assayMaterials therefor for pre-existing immune complex or autoimmune disease
  • A61K 31/664 - Amides of phosphorus acids
  • G16H 10/40 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for data related to laboratory analysis, e.g. patient specimen analysis

17.

AAV-CFTR VECTORS AND METHODS OF USING SAME

      
Application Number US2024033546
Publication Number 2024/258925
Status In Force
Filing Date 2024-06-12
Publication Date 2024-12-19
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Maeda, Yutaka
  • Brewington, John
  • Ostmann, Alicia

Abstract

Rhodothermus marinusRhodothermus marinus intein-N cDNA (CFTR-N-inteinN) and a second construct comprising a second portion of a human CFTR gene fused to Rhodothermus marinus intein-C cDNA (inteinC-CFTR-C). Further disclosed are methods of treating an individual having cystic fibrosis (CF) comprising administration of the paired nucleic acid constructs.

IPC Classes  ?

  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • C12N 15/861 - Adenoviral vectors

18.

SARS-COV 2 VACCINE COMPOSITIONS AND METHODS OF USING SAME

      
Application Number US2024033948
Publication Number 2024/259188
Status In Force
Filing Date 2024-06-14
Publication Date 2024-12-19
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor Tan, Ming

Abstract

Disclosed herein arc vaccine compositions, in particular, polyvalent icosahedral compositions for presentation of a SARS-CoV-2 antigen. The disclosed compositions may contain an S particle comprising a norovirus (NoV) S domain protein and a SARS-CoV-2 antigen, which may be linked via a linker protein domain operatively connected to the norovirus S domain protein and the SARS- CoV-2 antigen. Methods of using the disclosed vaccine composition are also provided.

IPC Classes  ?

19.

TREATMENT OF STEM CELL GRAFT FAILURE

      
Application Number US2024032938
Publication Number 2024/254401
Status In Force
Filing Date 2024-06-07
Publication Date 2024-12-12
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Sabulski, Anthony, Joseph
  • Jodele, Sonata

Abstract

Disclosed are methods for determining risk of transplant graft rejection of an individual, and treatment thereof. The method may comprise a) identifying a risk level of the individual as either high-risk or moderate-risk, the identification comprising determining the presence of a risk factor selected from mismatched or haploidentical donor, ex vivo T-cell depleted graft, and history of prior graft failure; and b) categorizing an individual with two identified risk factors as high-risk and an individual with one identified risk factor as moderate-risk. The method may further comprise administering an IFNy neutralizing agent to the individual identified as high-risk.

IPC Classes  ?

  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
  • C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies

20.

POLYPEPTIDES, NUCLEIC ACID MOLECULES, COMPOSITIONS, AND RELATED METHODS

      
Application Number 18786697
Status Pending
Filing Date 2024-07-29
First Publication Date 2024-12-05
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES (USA)
Inventor
  • Millay, Douglas
  • Gamage, Dilani
  • Chernomordik, Leonid
  • Leikina, Evgenia

Abstract

Some embodiments of the invention include polypeptides comprising a myomerger polypeptide or an extracellular myomerger polypeptide. Other embodiments of the invention include nucleic acid molecules encoding polypeptides comprising a myomerger polypeptide or an extracellular myomerger polypeptide. Other embodiments of the invention include vectors comprising the nucleic acid molecule. Yet other embodiments of the invention include methods of using a myomerger poly peptide or an extracellular myomerger polypeptide. Additional embodiments of the invention are also discussed herein.

IPC Classes  ?

  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • A61K 38/00 - Medicinal preparations containing peptides
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • C12N 5/16 - Animal cells

21.

Actor-Oriented Architecture Assessment Tool

      
Application Number 18673812
Status Pending
Filing Date 2024-05-24
First Publication Date 2024-11-28
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Seid, Michael
  • Hartley, David

Abstract

Disclosed herein are systems and methods for assessing a learning network (LN). Aspects may include collecting data related to an organization to be assessed, analyzing the data with respect to a set of indicators associated with an organizational ontology of an actor-oriented architecture (AOA), determining a strength of LN capabilities for each indicator in the set of indicators, generating a dashboard to graphically communicate at least one summary-statistic indicative of a strength of at least one LN capability.

IPC Classes  ?

22.

LIVER ORGANOIDS WITH INTRAHEPATIC SYMPATHETIC NERVES, AND METHODS OF USE THEREOF

      
Application Number US2024030068
Publication Number 2024/238977
Status In Force
Filing Date 2024-05-17
Publication Date 2024-11-21
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Takebe, Takanori
  • Ouchi, Rie

Abstract

Disclosed herein are methods of producing liver organoids with intrahepatic sympathetic neurons, or sympathetic nerves, from pluripotent stem cells. These intrahepatic sympathetic neurons, or sympathetic nerves, are involved in fatty liver disease pathogenesis, and the liver organoids produced from the methods provided herein may be used as a model system for fatty liver disease and pharmaceutical screening.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

23.

PROGNOSTIC AND PREDICTIVE VALUE OF ENDOTHELIAL DYSFUNCTION BIOMARKERS IN SEPSIS-ASSOCIATED ACUTE KIDNEY INJURY

      
Application Number US2024028949
Publication Number 2024/233965
Status In Force
Filing Date 2024-05-10
Publication Date 2024-11-14
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Atreya, Mihir
  • Stanski, Natalja

Abstract

Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in pediatric patients. Certain aspects of the invention relate to identifying one or more biomarkers associated with septic shock in pediatric patients in combination with one or more endothelial-derived biomarkers, receiving a dataset comprising biomarker concentrations, wherein the dataset is from a sample obtained from a pediatric patient having at least one indication of septic shock, then determining whether the biomarker concentrations of each of the at least one biomarkers are greater than one or more pre-determined cut-off biomarker concentration, wherein the level of said biomarker correlates with a predicted outcome.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems

24.

LIVER ORGANOID MODEL FOR HYPERBILIRUBINEMIA AND METHODS OF MAKING AND USING SAME

      
Application Number 18568764
Status Pending
Filing Date 2022-06-10
First Publication Date 2024-11-07
Owner CHILDREN’S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Takebe, Takanori
  • Reza, Hasan Al

Abstract

Disclosed herein are improved methods of maturing pluripotent stem cell-derived liver organoids with the use of hemoglobin metabolites such as bilirubin. Furthermore, by modulating the amounts of bilirubin used, liver organoids can be used as a model for hyperbilirubinemia. The scalability and tractability of these liver organoids made them excellent targets for drug screening against diseases such as hyperbilirubinemia. Also shown herein is the use of exogenous L-gulonolactone oxidase for improving viability of bilirubin-treated liver organoids.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

25.

ADVANCED MODELING FOR BIOLOGICS

      
Application Number 18654650
Status Pending
Filing Date 2024-05-03
First Publication Date 2024-11-07
Owner Children's Hospital Medical Center (USA)
Inventor Minar, Phillip

Abstract

Disclosed are methods for determining an initial or subsequent dose of an anti-tumor necrosis factor α (anti-TNFα) biologic for administering to an individual having an inflammatory condition, based on, in part, determining in the individual a level of one or more time-varying covariates selected from weight, albumin, erythrocyte sedimentation rate (ESR), neutrophil CD64 (nCD64) expression, and combinations thereof, and applying a correction factor to account for the changes in covariates over time. The inflammatory condition may be one selected from an IBD, such as Crohn's Disease (CD) or Ulcerative Colitis (UC), or inflammatory conditions such as uveitis or rheumatoid arthritis.

IPC Classes  ?

  • G16H 20/17 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients delivered via infusion or injection
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61P 1/00 - Drugs for disorders of the alimentary tract or the digestive system
  • A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
  • C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons

26.

COMPOSITIONS FOR INTERFERON BLOCKADE AND METHODS OF USING SAME

      
Application Number 18769116
Status Pending
Filing Date 2024-07-10
First Publication Date 2024-10-31
Owner Children's Hospital Medical Center (USA)
Inventor Jodele, Sonata

Abstract

Disclosed are compositions that may include one or more inhibitors of interferon activity for the treatment of a disease state, for example, a disorder associated with increased interferon levels such as thrombotic microangiopathy (“TMA”). Also disclosed are methods of treating an individual having a disease state such as thrombotic microangiopathy. Further disclosed are methods of diagnosing an individual with TMA.

IPC Classes  ?

  • C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61P 7/02 - Antithrombotic agentsAnticoagulantsPlatelet aggregation inhibitors
  • C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
  • C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

27.

METHODS AND SYSTEMS FOR TRAINING AND PREPARING MACHINE LEARNING MODEL(S) FOR USE IN IDENTIFYING A SPECIFIC PATIENT'S FUTURE RISK OF DEVELOPING A PSYCHIATRIC CONDITION

      
Application Number US2024025223
Publication Number 2024/220692
Status In Force
Filing Date 2024-04-18
Publication Date 2024-10-24
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Pestian, John
  • Glauser, Tracy
  • Santel, Daniel

Abstract

Early identification of psychiatric conditions such as anxiety and depression in children, adolescents, and young adults coupled with evidenced based treatment can significantly reduce acute and chronic morbidity and complications that occur later in life. An individual with a high risk of developing these serious psychiatric conditions can be identified with data and machine-learning methods before the conditions develop. A method for training and preparing a machine learning model for use in identifying a specific patient's future risk of developing a psychiatric condition (e.g. diagnosis, emergency room visit, hospitalization, suicide attempt) includes the following steps: collecting first electronic health records from previous patients who have been diagnosed with the psychiatric condition; processing the collected electronic health records into a training dataset and training a machine learning model with the training dataset to compute the specific patient's future risks for developing the psychiatric condition (e.g. diagnosis, emergency room visit, hospitalization, suicide attempt).

IPC Classes  ?

  • G16H 10/60 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for patient-specific data, e.g. for electronic patient records
  • G06N 20/00 - Machine learning
  • G16H 50/70 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for mining of medical data, e.g. analysing previous cases of other patients

28.

COMBINATION THERAPY FOR VAV3 CANCER

      
Application Number 18681723
Status Pending
Filing Date 2022-08-09
First Publication Date 2024-10-10
Owner
  • Children's Hospital Medical Center (USA)
  • UNIVERSITY OF CINCINNATI (USA)
Inventor
  • Nassar, Nicolas
  • Cancelas, Jose A.

Abstract

Disclosed herein are compositions and methods for treating an individual having a leukemia. In certain aspects, the methods may include administering an I0DVA1 compound and ponatinib to an individual in need thereof for treatment of a leukemia, which may include, for example, TKI-resistant leukemia, TKI-resistant Ph+ B-ALL, Chronic Myelogenous Leukemia (CML), Ph-Positive Acute Lymphoblastic Leukemia (ALL), Ph-like ALL, Resistant Chronic Phase Chronic Myeloid Leukemia (CP-CML), MLL-rearranged B-ALL, and VAV3 positive leukemia.

IPC Classes  ?

  • A61K 31/53 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
  • A61K 31/341 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
  • A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
  • A61K 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
  • A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
  • A61P 35/02 - Antineoplastic agents specific for leukemia

29.

CLINICAL-GRADE ORGANOIDS

      
Application Number US2024022355
Publication Number 2024/206911
Status In Force
Filing Date 2024-03-29
Publication Date 2024-10-03
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Kimura, Masaki
  • Takebe, Takanori

Abstract

Disclosed herein are improved methods of expanding cell populations and methods of producing organoids from pluripotent stem cells, as well as compositions and uses thereof, compositions including an amino acid supplemented liquid component which can be used for performing the methods, and kits including components for performing the methods and/or using the products of the methods. These methods may be performed without the need for xenogeneic and undefined basement membrane matrices for cell culture, which enables good manufacturing practice (GMP) compliant approaches for producing organoids for various uses such as clinical screening and therapeutics, including methods for treating a liver-related disease or disorder and screening candidate compounds or compositions for treating the same.

IPC Classes  ?

30.

STRUCTURALLY COMPLETE ORGANOIDS

      
Application Number 18573954
Status Pending
Filing Date 2022-06-30
First Publication Date 2024-09-26
Owner Children's Hospital Medical Center (USA)
Inventor
  • Wells, James Macormack
  • Eicher, Alexandra Kay

Abstract

Disclosed herein are compositions of gastrointestinal organoids comprising cells originating from all three primary germ layers and methods of making and use thereof. These gastrointestinal organoids exhibit complex cellular organization and functions resembling naturally occurring organ tissue, and serve as excellent three dimensional models for studying gastrointestinal physiology.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

31.

VASCULARIZED ORGANOIDS

      
Application Number 18684587
Status Pending
Filing Date 2022-08-17
First Publication Date 2024-09-26
Owner CHILDREN’S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Gu, Mingxia
  • Miao, Yifei

Abstract

Disclosed herein are compositions of organoids comprising a mesodermal vascular network and definitive endoderm derivatives. Also disclosed herein are methods making use of human pluripotent stem cells to differentiate cells of multiple germ layer lineages with proper organization. These organoids exhibit vasculature and can be used as models for studying endoderm-derived organogenesis and vascular interactions.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

32.

NOROVIRUS S PARTICLE BASED VACCINES AND METHODS OF MAKING AND USING SAME

      
Application Number 18490287
Status Pending
Filing Date 2023-10-19
First Publication Date 2024-09-26
Owner Children's Hospital Medical Center (USA)
Inventor
  • Tan, Ming
  • Jiang, Xi

Abstract

Disclosed herein are vaccine compositions, in particular, polyvalent icosahedral compositions for antigen presentation. The disclosed compositions may contain an S particle made up of recombinant fusion proteins. The recombinant fusion proteins may include a norovirus (NoV) S domain protein, a linker protein domain operatively connected to the norovirus S domain protein, and an antigen protein domain operatively connected to said linker.

IPC Classes  ?

  • A61K 39/12 - Viral antigens
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61K 39/385 - Haptens or antigens, bound to carriers
  • A61P 31/14 - Antivirals for RNA viruses
  • C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
  • C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof

33.

SYSTEMS AND METHODS FOR RESPIRATION-CONTROLLED VIRTUAL EXPERIENCES

      
Application Number 18732074
Status Pending
Filing Date 2024-06-03
First Publication Date 2024-09-26
Owner Children's Hospital Medical Center (USA)
Inventor
  • Hess, Abby Victoria
  • Lane, Blake

Abstract

Disclosed herein are systems and methods for respiration-controlled virtual experiences. In an embodiment, a controller presents a virtual experience via a user interface that is perceptible to a user. The controller receives a respiration-data stream from a respiration device with which the user is operably engaged. The respiration-data stream includes one or more respiration-parameter values of one or more respiration parameters associated with ongoing respiration of the user. The controller updates the virtual experience based at least in part on the one or more respiration-parameter values in the respiration-data stream. The virtual experience includes a plurality of sequential phases that have an associated phase sequence. Among the sequential phases is an active-induction phase that corresponds in time to the user receiving anesthetic induction via the respiration device, as well as one or more phases that each precede the active-induction phase.

IPC Classes  ?

  • A61B 5/087 - Measuring breath flow
  • A61B 5/00 - Measuring for diagnostic purposes Identification of persons
  • A61B 5/08 - Measuring devices for evaluating the respiratory organs
  • A61B 5/097 - Devices for facilitating collection of breath or for directing breath into or through measuring devices
  • A61B 5/16 - Devices for psychotechnicsTesting reaction times
  • A61M 16/01 - Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators Tracheal tubes specially adapted for anaesthetising
  • A61M 16/06 - Respiratory or anaesthetic masks
  • A61M 21/00 - Other devices or methods to cause a change in the state of consciousnessDevices for producing or ending sleep by mechanical, optical, or acoustical means, e.g. for hypnosis
  • A63F 13/21 - Input arrangements for video game devices characterised by their sensors, purposes or types
  • A63F 13/212 - Input arrangements for video game devices characterised by their sensors, purposes or types using sensors worn by the player, e.g. for measuring heart beat or leg activity
  • G06F 3/01 - Input arrangements or combined input and output arrangements for interaction between user and computer

34.

MODULATION OF ANTIBODY FUNCTION VIA SIALIC ACID MODIFICATION

      
Application Number 18279250
Status Pending
Filing Date 2022-03-04
First Publication Date 2024-09-19
Owner Children's Hospital Medical Center (USA)
Inventor
  • Way, Sing Sing
  • Erickson, John

Abstract

Disclosed are methods of modulating antibody function, in particular, via modifying one or more terminal sialic acid residues of an antibody. In certain aspects, the antibody may be an IgG antibody, wherein one or more terminal sialic acid residues is modified to modulate the antibody function. Further disclosed are methods of using such modified antibodies, and compositions comprising modified antibodies.

IPC Classes  ?

  • C07K 16/12 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from bacteria
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies

35.

MULTI-CYCLIC IRAK AND FLT3 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number 18258586
Status Pending
Filing Date 2021-12-23
First Publication Date 2024-09-05
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA,ASREPRESENTED BY THE SECRETARY,DEPT. OF HEALTH AND HUMAN SERVICES (USA)
Inventor
  • Hoyt, Scott Bryan
  • Thomas, Craig Joseph
  • Starczynowski, Daniel T.
  • Rosenbaum, Jan
  • Gracia Maldonado, Gavriel

Abstract

Some embodiments of the disclosure include inventive compounds (e.g., compounds of Formula (I)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.), Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.

IPC Classes  ?

  • C07D 487/04 - Ortho-condensed systems
  • A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
  • A61K 31/4985 - Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
  • A61K 31/541 - Non-condensed thiazines containing further heterocyclic rings
  • A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
  • A61P 35/02 - Antineoplastic agents specific for leukemia
  • C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or

36.

DIFFERENTIAL CXCR4 EXPRESSION ON HEMATOPOIETIC PROGENITOR CELLS VERSUS STEM CELLS DIRECTS HOMING AND LONG-TERM ENGRAFTMENT

      
Application Number 18578381
Status Pending
Filing Date 2022-07-12
First Publication Date 2024-09-05
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor Malik, Punam

Abstract

Compositions and methods for delivering a protein of interest such as a cell homing molecule (e.g., CXCR4) into host cells (e.g., to hematopoietic cells). The compositions and methods provided herein may be used to enhance homing and long-term engraftment of hematopoietic cells post transplantation.

IPC Classes  ?

  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
  • C07K 14/715 - ReceptorsCell surface antigensCell surface determinants for cytokinesReceptorsCell surface antigensCell surface determinants for lymphokinesReceptorsCell surface antigensCell surface determinants for interferons
  • C12N 5/0789 - Stem cellsMultipotent progenitor cells
  • C12N 15/86 - Viral vectors

37.

Aldehyde dehydrogenase producing bacteria and methods of using same

      
Application Number 18625448
Grant Number 12138299
Status In Force
Filing Date 2024-04-03
First Publication Date 2024-08-29
Grant Date 2024-11-12
Owner Children's Hospital Medical Center (USA)
Inventor Alenghat, Theresa

Abstract

Disclosed herein are methods for enhancing an immune response in an individual in need thereof. The methods, in certain aspects, may comprise administering bacterial aldehyde dehydrogenase, a bacteria that produces aldehyde dehydrogenase, or combinations thereof to an individual. Further disclosed are compositions, such as nutritional compositions, which may comprise bacterial aldehyde dehydrogenase, a bacteria that produces aldehyde dehydrogenase, or combinations thereof.

IPC Classes  ?

38.

COMPOSITIONS AND METHODS FOR MITIGATING EPITHELIAL BARRIER DYSFUNCTION

      
Application Number US2024015947
Publication Number 2024/173651
Status In Force
Filing Date 2024-02-15
Publication Date 2024-08-22
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Hershey, Gurjit Khurana
  • Devore, Stanley

Abstract

The invention provides methods for treating a disease or disorder characterized by epithelial barrier dysfunction by modulating CARD14:MYC interaction, and related methods and compositions for modulating MYC activity in target epithelial cells, the methods comprising contacting the epithelial cells with an inhibitor or an agonist of CARD14 binding to MYC in the target epithelial cells.

IPC Classes  ?

  • A61K 31/426 - 1,3-Thiazoles
  • A61K 38/00 - Medicinal preparations containing peptides
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61P 17/06 - Antipsoriatics
  • A61P 37/08 - Antiallergic agents
  • C07D 277/00 - Heterocyclic compounds containing 1,3-thiazole or hydrogenated 1,3-thiazole rings
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

39.

MULTI-CYCLIC IRAK AND FLT3 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number US2024014038
Publication Number 2024/163764
Status In Force
Filing Date 2024-02-01
Publication Date 2024-08-08
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES (USA)
  • KUROME THERAPEUTICS, INC. (USA)
Inventor
  • Hoyt, Scott Bryan
  • Starczynowski, Daniel T.
  • Thomas, Craig Joseph
  • Rosenbaum, Jan Susan

Abstract

Some embodiments of the invention include inventive compounds (e.g., compounds of Formula (I), (II), or (III)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.

IPC Classes  ?

  • C07D 487/04 - Ortho-condensed systems
  • A61K 31/437 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
  • A61K 31/445 - Non-condensed piperidines, e.g. piperocaine
  • A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
  • A61K 31/4985 - Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
  • A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
  • A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings

40.

MODEL-INFORMED PRECISION DOSING SYSTEMS FOR TREATMENT OF INFLAMMATORY BOWEL DISEASE

      
Application Number 18413823
Status Pending
Filing Date 2024-01-16
First Publication Date 2024-07-18
Owner Children's Hospital Medical Center (USA)
Inventor
  • Minar, Phillip
  • Vinks, Alexander

Abstract

Disclosed are methods for preparing a patient-specific vedolizumab dosing regimen for an individual in need thereof, comprising accessing, using a processor, a memory; selecting a first model stored in the memory; forecasting, based on the selected first model, a patient-specific predicted concentration time profile of the vedolizumab, selecting a dosing regimen, based on said forecasting, wherein said dosing regimen achieves a treatment objective, said treatment objective being a therapeutic level of vedolizumab; and outputting said dosing regimen. Further disclosed are methods of treating an individual having an inflammatory bowel disease.

IPC Classes  ?

  • G16H 20/10 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients

41.

COLONIC ORGANOIDS AND METHODS OF MAKING AND USING SAME

      
Application Number 18473619
Status Pending
Filing Date 2023-09-25
First Publication Date 2024-07-18
Owner Children's Hospital Medical Center (USA)
Inventor
  • Wells, James M.
  • Munera, Jorge Orlando

Abstract

Disclosed herein are methods for the in vitro differentiation of a precursor cell into definitive endoderm, which may further be differentiated into a human colonic organoid (HCO), via modulation of signaling pathways. Further disclosed are HCOs and methods of using HCOs, which may be used, for example, for the HCOs may be used to determine the efficacy and/or toxicity of a potential therapeutic agent for a disease selected from colitis, colon cancer, polyposis syndromes, and/or irritable bowel syndrome.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

42.

INFLUENZA VACCINE COMPOSITIONS AND METHODS OF USING SAME

      
Application Number 18276905
Status Pending
Filing Date 2022-02-16
First Publication Date 2024-07-11
Owner Children's Hospital Medical Center (USA)
Inventor
  • Tan, Ming
  • Jiang, Xi Jason

Abstract

The instant disclosure relates to pseudovirus nanoparticles (PVNPs) and compositions comprising PVNPs. The disclosed PVNPs may be comprised of fusion proteins that form an icosahedral structure and a nanoparticle shell. The disclosed fusion proteins may comprise a modified norovirus (NoV) S domain protein; a hemagglutinin I (HA1) antigen of the influenza hemagglutinin I (HA1) of influenza vims; and a peptide linker connecting the C-terminus of the NoV S domain to the HA1 antigen. The modified NoV S domain proteins form the interior nanoparticle shell of said PVNP composition and display the 60 HA1 antigens on the surface of the nanoparticle shell. Methods of making and using the PVNPs and compositions containing PVNPs are also disclosed.

IPC Classes  ?

  • A61K 39/145 - Orthomyxoviridae, e.g. influenza virus
  • A61P 31/16 - Antivirals for RNA viruses for influenza or rhinoviruses
  • C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
  • C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof

43.

CHIMERIC POLYPEPTIDES, NUCLEIC ACID MOLECULES, CELLS, AND RELATED METHODS

      
Application Number 17052894
Status Pending
Filing Date 2019-05-06
First Publication Date 2024-07-04
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Waggoner, Stephen
  • Brunner, Hermine
  • Reighard, Seth

Abstract

Some embodiments of the invention include chimeric polypeptides. Other embodiments of the invention include chimeric nucleic acid molecules encoding a chimeric polypeptide. Other embodiments of the invention include vectors comprising a chimeric nucleic acid molecule. Still other embodiments of the invention include cells comprising a chimeric nucleic acid molecule, a chimeric polypeptide, or both. Yet other embodiments of the invention include methods of making cells. Some embodiments include methods of treating disease. Additional embodiments of the invention are also discussed herein.

IPC Classes  ?

44.

RESORBABLE MATERIAL ACTIVATED MECHANISMS

      
Application Number 18567708
Status Pending
Filing Date 2022-06-07
First Publication Date 2024-06-27
Owner
  • Children's Hospital Medical Center (USA)
  • Brigham Young University (USA)
Inventor
  • Parkinson, Bethany M.
  • Sargent, Brandon
  • Garcia, Victor
  • Howell, Larry L.

Abstract

A device using resorbable materials to actuate, such as a device (10) for repairing pectus excavatum in a patient through activation of a bioresorbable material (40, 52).

IPC Classes  ?

  • A61B 17/80 - Cortical plates
  • A61B 17/00 - Surgical instruments, devices or methods
  • A61B 17/56 - Surgical instruments or methods for treatment of bones or jointsDevices specially adapted therefor

45.

A CONTINUOUS METRIC TO MEASURE ENDOTYPE AND CORTICOSTEROID INTERACTION IN SEPTIC SHOCK

      
Application Number 18264741
Status Pending
Filing Date 2022-02-16
First Publication Date 2024-06-27
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • VANDERBILT UNIVERSITY (USA)
Inventor
  • Wong, Hector
  • Lindsell, Christopher

Abstract

Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in patients, such as pediatric. In particular, the invention relates to analyzing biomarkers associated with septic shock in pediatric patients, obtaining a sample from a patient having at least one indication of septic shock, then determining the gene expression mosaic for the patient, wherein the gene expression mosaic can correlate with a predicted outcome and can inform treatment strategy.

IPC Classes  ?

  • C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
  • A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • G16H 10/40 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for data related to laboratory analysis, e.g. patient specimen analysis
  • G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment

46.

IMPROVED ECULIZUMAB PRECISION DOSING

      
Application Number US2023085290
Publication Number 2024/137904
Status In Force
Filing Date 2023-12-21
Publication Date 2024-06-27
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Jodele, Sonata
  • Mizuno, Kana
  • Vinks, Alexander, A.

Abstract

Disclosed herein are methods of treating a human patient having TA-TMA, for example high- risk TA-TMA with MODS, comprising administration of a C5 inhibitor, for example eculizumab or an antigen binding fragment thereof. The disclosed dosing regimens may comprise one or more of a loading phase, induction phase, and maintenance phase. The disclosed methods are particularly advantageous for individuals that do not have clinically relevant bleeding.

IPC Classes  ?

  • C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
  • A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid

47.

METHOD OF MAKING IN VIVO HUMAN SMALL INTESTINE ORGANOIDS FROM PLURIPOTENT STEM CELLS

      
Application Number 18156726
Status Pending
Filing Date 2024-03-08
First Publication Date 2024-06-20
Owner Children's Hospital Medical Center (USA)
Inventor
  • Wells, James M.
  • Watson, Carey Lane
  • Munera, Jorge Orlando
  • Mahe, Maxime Mickael
  • Helmrath, Michael A.
  • Workman, Michael J.

Abstract

Disclosed are methods for making a vascularized hollow organ derived from human intestinal organoid (HIOs). The HIOs may be obtained from human embryonic stem cells (ESC's) and/or induced pluripotent stem cells (iPSCs), such that the HIO forms mature intestinal tissue. Also disclosed are methods for making a human intestinal tissue containing a functional enteric nervous system (ENS).

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • A01K 67/0271 - Chimeric vertebrates, e.g. comprising exogenous cells
  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing

48.

USE OF 6-THIO-dG TO TREAT THERAPY-RESISTANT TELOMERASEPOSITIVE PEDIATRIC BRAIN TUMORS

      
Application Number 18511417
Status Pending
Filing Date 2023-11-16
First Publication Date 2024-06-13
Owner
  • The Board of Regents of The University of Texas System (USA)
  • Children's Hospital Medical Center (USA)
Inventor
  • Shay, Jerry
  • Drissi, Rachid

Abstract

Brain tumors remain the leading cause of cancer-related deaths in children and often are associated with long-term sequelae among survivors of current therapies. Telomerase and telomeres play important roles in cancer, representing attractive therapeutic targets to treat children with poor-prognosis brain tumors such as diffuse intrinsic pontine glioma (DIPG), high-grade glioma (HGG) and high-risk medulloblastoma (MB). It has shown that DIPG, HGG and MB frequently express telomerase activity. It is now shown that the telomerase-dependent incorporation of 6-thio-2′deoxyguanosine (6-thio-dG), a telomerase substrate precursor analog, into telomeres leads to telomere dysfunction-induced foci (TIFs) along with extensive genomic DNA damage, cell growth inhibition and cell death of primary stem-like cells derived from patients with DIPG, HGG and MB. Importantly, the effect of 6-thio-dG is persistent even after drug withdrawal. Treatment with 6-thio-dG elicits a sequential activation of ATR and ATM pathways and induces G2/M arrest. In vivo, treatment of mice bearing MB xenografts with 6-thio-dG delays tumor growth, increases in-tumor TIFs and apoptosis. Furthermore, 6-thio-dG crosses the blood-brain barrier and specifically targets tumor cells in an orthotopic mouse model of DIPG. Together, these findings suggest that 6-thio-dG is a promising approach to treat therapy-resistant telomerase-positive pediatric brain tumors.

IPC Classes  ?

  • A61K 31/708 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid having oxo groups directly attached to the purine ring system, e.g. guanosine, guanylic acid
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 35/00 - Antineoplastic agents

49.

ACCESS PORT ASSEMBLY, FLOW CONTROL SYSTEM AND TRAJECTORY DETERMINATION ASSEMBLY, AND METHODS FOR USING THE AFOREMENTIONED

      
Application Number IB2023062295
Publication Number 2024/121767
Status In Force
Filing Date 2023-12-06
Publication Date 2024-06-13
Owner
  • B. G. NEGEV TECHNOLOGIES AND APPLICATIONS LTD., AT BEN-GURION UNIVERSITY (Israel)
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Mangano, Francesco
  • Zarrouk, David
  • Daniel, Atai
  • Peer, Segev
  • Grinshpan, Ben
  • Coronel, Matan

Abstract

Embodiments pertain to an access port assembly configured to assist in creating access for and accommodating a medical device extending from outside a patient body and terminating in a cavity internal to the patient body. The assembly may comprise a guide element comprising a guide channel having a longitudinal guide axis extending from an inlet to an outlet of the guide element for guiding the medical device through the guide channel. The guide element may be movably coupled with the fixation base by an articulating linkage to allow selecting an orientation of the guide channel relative to the site of interest of the patient body. The articulating linkage may be implemented as a ball-and-socket linkage. Embodiments also pertain to a system for controlling delivery of fluid into the patient body and/or removal of fluid from the patient body, via one or more access ports.

IPC Classes  ?

  • A61B 90/11 - Instruments, implements or accessories specially adapted for surgery or diagnosis and not covered by any of the groups , e.g. for luxation treatment or for protecting wound edges for stereotaxic surgery, e.g. frame-based stereotaxis with guides for needles or instruments, e.g. arcuate slides or ball joints
  • A61B 17/34 - TrocarsPuncturing needles
  • F16M 11/14 - Means for attachment of apparatusMeans allowing adjustment of the apparatus relatively to the stand allowing pivoting in more than one direction with ball-joint

50.

NON-INVASIVE METHODS FOR SKIN SAMPLE COLLECTION AND ANALYSIS

      
Application Number 18435217
Status Pending
Filing Date 2024-02-07
First Publication Date 2024-05-30
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Hershey, Gurjit Khurana
  • Biagini-Myers, Jocelyn
  • Schauberger, Eric

Abstract

Kits for assessing a skin condition of a subject comprise (a) a first set of adhesive tapes adapted to adhere a first biological material from stratum corneum of a target skin site of the subject, (b) a second set of adhesive tapes adapted to adhere a second biological material from an epidermis layer below stratum corneum of a target skin site of the subject, wherein each of the adhesive tapes in the first set of adhesive tapes and the second set of adhesive tapes comprises a water-soluble adhesive layer attached to a substrate, (c) one or more reagents for extracting biological material adhered to the water-soluble adhesive layers of the adhesive tapes, and (d) one or more detection agents for determining a presence of microbial biological materials and/or host biological materials from water-soluble adhesive tape extract.

IPC Classes  ?

  • A61B 10/02 - Instruments for taking cell samples or for biopsy
  • C12Q 1/6888 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for detection or identification of organisms

51.

SYSTEMS AND METHODS FOR ENHANCED ENCODED SOURCE IMAGING

      
Application Number 18422349
Status Pending
Filing Date 2024-01-25
First Publication Date 2024-05-23
Owner Children's Hospital Medical Center (USA)
Inventor Xiang, Jing

Abstract

A frequency encoded source imaging system includes an EEG or MEG sensor array and a processing system for analyzing the signals from the sensor array in at least two different frequency bands, where the analysis is localized with respect to a three-dimensional grid corresponding to the portion of the human body. Alternately, a frequency encoded source imaging system includes an EEG or MEG sensor array and a processing system for analyzing the signals from the sensor array in a high-definition frequency band comprising frequencies greater than 70 Hz, where the analysis is localized with respect to a three-dimensional grid corresponding to the portion of the human body

IPC Classes  ?

  • A61B 5/374 - Detecting the frequency distribution of signals, e.g. detecting delta, theta, alpha, beta or gamma waves
  • A61B 5/00 - Measuring for diagnostic purposes Identification of persons
  • A61B 5/055 - Detecting, measuring or recording for diagnosis by means of electric currents or magnetic fieldsMeasuring using microwaves or radio waves involving electronic [EMR] or nuclear [NMR] magnetic resonance, e.g. magnetic resonance imaging
  • A61B 5/245 - Detecting biomagnetic fields, e.g. magnetic fields produced by bioelectric currents specially adapted for magnetoencephalographic [MEG] signals
  • A61B 6/46 - Arrangements for interfacing with the operator or the patient

52.

VP4-BASED TRIVALENT PSEUDOVIRUS NANOPARTICLE VACCINE FOR ROTAVIRUS AND METHODS OF USING SAME

      
Application Number US2023078895
Publication Number 2024/102697
Status In Force
Filing Date 2023-11-07
Publication Date 2024-05-16
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor Tan, Ming

Abstract

Disclosed herein are vaccine compositions, in particular, polyvalent icosahedral compositions for presentation of a rotavirus antigen. The disclosed compositions may contain an S particle made up of recombinant fusion proteins that further comprise a rotavirus antigen. The recombinant fusion proteins may include a norovirus (NoV) S domain protein, a linker protein domain operatively connected to the norovirus S domain protein, and a rotavirus antigen protein domain. The disclosed particles and compositions may be used as a vaccine composition for reducing the likelihood of becoming infected with rotavirus, diminishing the severity of a rotavirus infection, reducing the duration of time of a rotavirus infection, or otherwise improving an immune response following contact with rotavirus in an individual.

IPC Classes  ?

  • A61K 39/12 - Viral antigens
  • A61K 39/385 - Haptens or antigens, bound to carriers
  • A61P 31/14 - Antivirals for RNA viruses
  • C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses

53.

MACHINE LEARNING DRIVEN IDENTIFICATION OF GENE-EXPRESSION SIGNATURES ASSOCIATED WITH PERSISTENT MULTIPLE ORGAN DYSFUNCTION

      
Application Number US2023078012
Publication Number 2024/092190
Status In Force
Filing Date 2023-10-27
Publication Date 2024-05-02
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • EMORY UNIVERSITY (USA)
Inventor
  • Atreya, Mihir
  • Kamaleswaran, Rishikesan

Abstract

Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in pediatric patients. Certain aspects of the disclosure relates to identifying one or more biomarkers associated with septic shock in pediatric patients in combination with one or more endothelial-derived biomarkers, obtaining a sample from a pediatric patient having at least one indication of septic shock, then quantifying from the sample an amount of said biomarkers, wherein the level of said biomarker correlates with a predicted outcome.

IPC Classes  ?

  • A61B 5/00 - Measuring for diagnostic purposes Identification of persons
  • C12Q 1/68 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving nucleic acids
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing

54.

SYNTHETIC AMNIOTIC FLUID COMPOSITIONS COMPRISING URINARY TRYPSIN INHIBITOR AND ASCORBIC ACID, AND METHODS OF USING SAME

      
Application Number US2023035776
Publication Number 2024/091484
Status In Force
Filing Date 2023-10-24
Publication Date 2024-05-02
Owner
  • CHILDERN'S HOSPITAL MEDICAL CENTER (USA)
  • UNIVERSITY OF CINCINNATI (USA)
Inventor
  • Oria, Marc
  • Forde, Braxton
  • Peiro-Ibanez, Jose Luis

Abstract

Disclosed are synthetic amniotic fluid compositions comprising ulin-A- statin/urinary trypsin inhibitor (UTI) and ascorbic acid and methods of using the disclosed synthetic amniotic fluid compositions, which may be useful for a variety of purposes related to treatment of a fetus, replacement of amniotic fluid, or research uses in which amniotic cells or tissues are used. The synthetic amniotic fluid compositions may be used to minimize and/or reduce risks associated with currently utilized compositions for amnioinfusion, which traditionally include normal saline (NS) and Lactated Ringer's (LR), or to improve amniotic epithelial cell viability and integrity, in vivo and/or in vitro.

IPC Classes  ?

  • A61K 38/57 - Protease inhibitors from animalsProtease inhibitors from humans
  • A61K 31/375 - Ascorbic acid, i.e. vitamin CSalts thereof
  • A61K 31/7004 - Monosaccharides having only carbon, hydrogen and oxygen atoms
  • A61K 33/06 - Aluminium, calcium or magnesiumCompounds thereof
  • A61K 33/20 - Elemental chlorineInorganic compounds releasing chlorine
  • A61K 33/30 - ZincCompounds thereof
  • A61K 38/38 - Albumins
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 15/06 - Antiabortive agentsLabour repressants
  • A61P 43/00 - Drugs for specific purposes, not provided for in groups

55.

VLP-BASED BIVALENT EBOLA VACCINES AND METHODS OF MAKING AND USING SAME

      
Application Number 18392463
Status Pending
Filing Date 2023-12-21
First Publication Date 2024-04-25
Owner Children's Hospital Medical Center (USA)
Inventor
  • Singh, Karnail
  • Spearman, Paul

Abstract

Disclosed herein are virus-like particle (VLP)-based bivalent vaccine compositions. The compositions may comprise a spherical retroviral Group-specific Antigen (“Gag”) protein core and at least two Ebola glycoproteins. The at least two Ebola glycoproteins may be located at the exterior surface of the spherical Gag protein core, such that the VLP-based vaccine presents at least two Ebola glycoprotein antigens. In one aspect, the at least two Ebola glycoproteins are a Zaire (EBOV) glycoprotein, and a Sudan (SUDV) glycoprotein.

IPC Classes  ?

  • A61K 39/12 - Viral antigens
  • C07K 14/08 - RNA viruses
  • C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof

56.

DOSING ALGORITHM FOR COMPLEMENT INHIBITOR

      
Application Number 18499353
Status Pending
Filing Date 2023-11-01
First Publication Date 2024-04-18
Owner Children's Hospital Medical Center (USA)
Inventor
  • Jodele, Sonata
  • Fukuda, Tsuyoshi
  • Mizuno, Kana

Abstract

Described are methods and systems for the treatment of individuals having a disorder characterized by complement system dysregulation. The described methods and systems may be used for a variety of purposes, including for example, establishing one or both of a general or personalized dosing schedule for treatment using a complement inhibitor, establishing a dosage schedule sufficient to maintain an effective amount of complement inhibitor, establishing general dosing schedules for novel complement modifying agents and identifying a treatment regimen and/or dose eliminating the possibility of under dosing medication, and treatment regimen and/or dose for reducing or preventing toxicity in a patient.

IPC Classes  ?

  • C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
  • A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
  • A61P 37/02 - Immunomodulators
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • G16H 20/17 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients delivered via infusion or injection

57.

MULTI-CYCLIC IRAK AND FLT3 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number 18257746
Status Pending
Filing Date 2021-12-15
First Publication Date 2024-04-18
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • The United States of America, as Represented by the Secretary, Dept. of Health and Human Services (USA)
  • KUROME THERAPEUTICS, INC. (USA)
Inventor
  • Hoyt, Scott Bryan
  • Thomas, Craig Joseph
  • Starczynowski, Daniel T.
  • Sutter, Patrick Joseph
  • Tawa, Gregory James
  • Finocchio, Chris James
  • Rosenbaum, Jan Susan
  • Gracia Maldonado, Gabriel

Abstract

Some embodiments of the disclosure include inventive compounds (e.g., compounds of Formula (I)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.), Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.

IPC Classes  ?

  • C07D 487/04 - Ortho-condensed systems
  • A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
  • A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
  • A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
  • A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
  • A61P 35/02 - Antineoplastic agents specific for leukemia
  • C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or

58.

MULTI-CYCLIC IRAK AND FLT3 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number US2023034438
Publication Number 2024/076614
Status In Force
Filing Date 2023-10-04
Publication Date 2024-04-11
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVIC (USA)
  • KUROME THERAPEUTICS, INC. (USA)
Inventor
  • Hoyt, Scott, Bryan
  • Starczynowski, Daniel, T.
  • Thomas, Craig, Joseph
  • Rosenbaum, Jan, Susan

Abstract

The present disclosure provides pyrazolo[1,5-a]pyrimidine compounds and compositions comprising the same which inhibit IRAK and/or FLT3. The present disclosure further provides methods of using the pyrazolo[1,5-a]pyrimidine compounds to treat a disease or disorder such as a hematopoietic cancer, myelodysplastic syndromes (MDS), or acute myeloid leukemia (AML). Additional embodiments provide disease treatment using combinations of the disclosed IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.

IPC Classes  ?

  • A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
  • C07D 487/04 - Ortho-condensed systems

59.

ORGANOID COMPOSITIONS HAVING IMMUNE CELLS

      
Application Number US2023032650
Publication Number 2024/063999
Status In Force
Filing Date 2023-09-13
Publication Date 2024-03-28
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Helmrath, Michael, A.
  • Bouffi, Carine

Abstract

Disclosed herein are methods of generating intestinal organoids (IO) with immune cells (iIO) and methods of making an iIO with activated immune cells. IO compositions including immune cells, optionally including activated immune cells, (iIO) are also disclosed herein. Also disclosed are iIO models of disease states. Further disclosed are methods of treatment including transplanting iIO into an organism, as well as methods of screening a compound for activity using an iIO, for example an iIO model of a disease state.

IPC Classes  ?

  • A61L 27/38 - Animal cells
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing
  • A61K 39/108 - EscherichiaKlebsiella

60.

SERUM PROTEIN BIOMARKER PANEL FOR IDIOPATHIC PULMONARY FIBROSIS

      
Application Number 17766556
Status Pending
Filing Date 2020-10-08
First Publication Date 2024-03-28
Owner
  • Children's Hospital Medical Center (USA)
  • University of Michigan (USA)
Inventor
  • Hardie, William D.
  • Ziady, Assem
  • Moore, Bethany

Abstract

The instant disclosure relates to methods for assessing pulmonary fibrosis disorder disease status in an individual in need thereof. One aspect of the disclosed methods may comprise: detecting a level of one or more biomarkers in a biological sample obtained from an individual, comparing the level of the one or more biomarkers to that of a control value corresponding to the one or more biomarkers, characterizing the disease status in the individual based on the level of the one or more biomarkers as compared to that of a relevant control value; and administering a treatment to said individual based on the assessment of the one or more biomarker levels.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

61.

SELF-EXPANDABLE SURGICAL IMPLANT FOR CORRECTON OF CONGENITAL DIAPHRAGMATIC HERNIA

      
Application Number 18513858
Status Pending
Filing Date 2023-11-20
First Publication Date 2024-03-21
Owner Children's Hospital Medical Center (USA)
Inventor
  • Peiro Ibanez, Jose
  • Abello Munarriz, Cristobal

Abstract

A self-expandable double-sided surgical implant including a peripheral pocket receiving an expansion ring. The surgical implant may be used for the correction of congenital diaphragmatic hernias.

IPC Classes  ?

  • A61F 2/00 - Filters implantable into blood vesselsProstheses, i.e. artificial substitutes or replacements for parts of the bodyAppliances for connecting them with the bodyDevices providing patency to, or preventing collapsing of, tubular structures of the body, e.g. stents

62.

NUCLEIC ACID MOLECULES AND THEIR METHODS OF USE

      
Application Number 18507035
Status Pending
Filing Date 2023-11-11
First Publication Date 2024-03-21
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor Pan, Dao

Abstract

Some embodiments of the invention include a nucleic acid molecule comprising natural nucleotides, non-natural nucleotides, an LNA which comprises one or more RNA core molecules, or an RNA molecule which comprises more than one RNA core molecule. Some embodiments of the invention include a nucleic acid molecule comprising an RNA molecule which comprises more than one RNA core molecule. Other embodiments of the invention include a nucleic acid molecule comprising a DNA molecule encoding the RNA molecule (e.g., vector or viral vector). Other embodiments include compositions or pharmaceutical compositions that comprise the nucleic acid molecule. Some embodiments of the invention comprise reducing miR-143 in a cell. Other embodiments of the invention include methods to deliver a protein across the BBB. Other embodiments include methods for treating disease (e.g., LSD), neuronopathic disease, neurodegenerative disease, Hurler syndrome, or MPS I). Additional embodiments of the invention are also discussed herein.

IPC Classes  ?

  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • A61P 35/00 - Antineoplastic agents

63.

Microspheres Containing Decellularized Donor Tissue and Their Use in Fabricating Polymeric Structures

      
Application Number 18333934
Status Pending
Filing Date 2023-06-13
First Publication Date 2024-03-07
Owner
  • UNIVERSITY OF CINCINNATI (USA)
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Lin, Chia-Ying James
  • Gruber, Stacey
  • Whitlock, Patrick W.
  • Ghosh, Paulomi

Abstract

Decellularized matrix microspheres comprising a polymeric material and a donor tissue are provided. Also disclosed are structures containing a plurality of decellularized matrix microspheres incorporating a first polymer and a donor tissue; and a second polymer, wherein the decellularized matrix microspheres and the second polymer are in the form of a filament. Methods of treating a tissue injury employing the matrix microspheres and structures described as well as their methods of manufacture are also provided.

IPC Classes  ?

  • A61L 27/18 - Macromolecular materials obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds
  • A61L 27/36 - Materials for prostheses or for coating prostheses containing ingredients of undetermined constitution or reaction products thereof
  • B29C 64/118 - Processes of additive manufacturing using only liquids or viscous materials, e.g. depositing a continuous bead of viscous material using filamentary material being melted, e.g. fused deposition modelling [FDM]
  • B33Y 70/00 - Materials specially adapted for additive manufacturing
  • B33Y 70/10 - Composites of different types of material, e.g. mixtures of ceramics and polymers or mixtures of metals and biomaterials
  • B33Y 80/00 - Products made by additive manufacturing

64.

ADVANCED ALVEOLAR EPITHELIAL ORGANOIDS

      
Application Number US2023031312
Publication Number 2024/049773
Status In Force
Filing Date 2023-08-28
Publication Date 2024-03-07
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Zacharias, William
  • Toth, Andrea

Abstract

in vivoin vivo lung epithelial regeneration, providing a tractable model to dissect regenerative processes. Clonal expansion of single AEPs generated complex alveolar organoids with extensive internal organization. Whole mount immunohistochemistry of these organoids showed properly patterned, polarized, and functional AT1 and AT2 cells surrounding numerous alveolar-like cavities with minimal structural contribution from mesenchymal cells, implying extensive cell autonomous regenerative function encoded in adult AEPs.

IPC Classes  ?

  • A61K 35/42 - Respiratory system, e.g. lungs, bronchi or lung cells
  • C12N 5/02 - Propagation of single cells or cells in suspensionMaintenance thereofCulture media therefor
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • A61P 11/00 - Drugs for disorders of the respiratory system
  • C12N 5/074 - Adult stem cells

65.

METHODS FOR OPTIMIZING CFTR-MODULATOR THERAPY

      
Application Number 18272176
Status Pending
Filing Date 2022-01-14
First Publication Date 2024-02-29
Owner Children's Hospital Medical Center (USA)
Inventor
  • Ziady, Assem G.
  • Skala, Emily J.
  • Dunn, Maureen B.
  • Lammers, Karen

Abstract

Disclosed are methods for the treatment of cystic fibrosis in an individual in need thereof. The methods may include the administration of a CFTR modulator, with or without and one or more CFTR modulator therapy optimizing agent. Further disclosed are methods for treating cystic fibrosis in an individual in need thereof which employ detection of one or more biomarkers which may be used to distinguish CFTR modulator responders and non-responders, which may in turn be used to direct therapy in an individual having cystic fibrosis.

IPC Classes  ?

  • A61K 31/47 - QuinolinesIsoquinolines
  • A61K 31/4025 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil not condensed and containing further heterocyclic rings, e.g. cromakalim
  • A61K 31/44 - Non-condensed pyridinesHydrogenated derivatives thereof
  • A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
  • C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

66.

COMPOSITIONS AND METHODS FOR TREATMENT OF PEDIATRIC PAIN

      
Application Number 18380877
Status Pending
Filing Date 2023-10-17
First Publication Date 2024-02-08
Owner Children's Hospital Medical Center (USA)
Inventor
  • Jankowski, Michael P.
  • Liu, Xiaohua
  • Rose, John Barns

Abstract

Disclosed are methods of treating pain in a mammal, which may include the step of administering human growth hormone to a mammal in need thereof. The pain treated by the disclosed methods may be of a type caused by inflammation induced mechanical and/or thermal hypersensitivity, and may include, for example, a pain type resulting from one or more conditions selected from peripheral injury pain, post-operative pain, cutaneous inflammation, cutaneous incision, muscle incision, or chronic pain. Disease states in which the disclosed methods may be used include fibromyalgia, sickle cell anemia, epidermolysis bullosa, erythromelalgia, complex regional pain syndrome, or generalized muscle pain.

IPC Classes  ?

  • A61K 38/27 - Growth hormone [GH], i.e. somatotropin

67.

MULTI-CYCLIC IRAK1 AND IRAK4 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number US2023071435
Publication Number 2024/030908
Status In Force
Filing Date 2023-08-01
Publication Date 2024-02-08
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVIC (USA)
  • KUROME THERAPEUTICS, INC. (USA)
Inventor
  • Thomas, Craig, Joseph
  • Hoyt, Scott, Bryan
  • Starczynowski, Daniel, T.
  • Rosenbaum, Jan, Susan
  • Bennett, Joshua

Abstract

The present disclosure provides a method of treating an inflammatory disease/disorder, acute myeloid leukemia (AML), or myelodysplastic syndrome (MDS) in a subject comprising administering to the subject a compound that inhibits IRAKI and IRAK4. The present disclosure further provides a method of determining a compound that is effective at treating an inflammatory disease/disorder, AML, or MDS.

IPC Classes  ?

  • C07D 471/02 - Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups in which the condensed system contains two hetero rings
  • A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
  • A61P 35/00 - Antineoplastic agents
  • C07D 513/22 - Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups , or in which the condensed system contains four or more hetero rings

68.

SYSTEM AND METHOD FOR GUIDING AN INVASIVE DEVICE

      
Application Number 18483729
Status Pending
Filing Date 2023-10-10
First Publication Date 2024-02-01
Owner Children's Hospital Medical Center (USA)
Inventor
  • Dumoulin, Charles Lucian
  • Johnson, Neil David
  • Pratt, Ronald Gene

Abstract

A system for guiding a medical intervention is disclosed. The system employs a device guide that operates on the surface of a sphere that is centered on a selected target.

IPC Classes  ?

  • G01R 33/28 - Details of apparatus provided for in groups
  • A61B 90/00 - Instruments, implements or accessories specially adapted for surgery or diagnosis and not covered by any of the groups , e.g. for luxation treatment or for protecting wound edges
  • A61B 17/34 - TrocarsPuncturing needles
  • G01R 33/50 - NMR imaging systems based on the determination of relaxation times

69.

POPULATION-SCALE ORGANOID POOLS

      
Application Number US2023028409
Publication Number 2024/025808
Status In Force
Filing Date 2023-07-21
Publication Date 2024-02-01
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Takebe, Takanori
  • Kimura, Masaki

Abstract

Disclosed are improved methods of making liver organoids, and methods of making population organoid panels which can be used, for example, for genotype-pheontype analysis or screening of test compounds. Also disclosed are methods of assessing the risk/prognosis of fatty acid liver disease in subjects having a SNP variant GCKR-rs1260326, as well as novel methods of treating such subjects.

IPC Classes  ?

  • C12N 5/02 - Propagation of single cells or cells in suspensionMaintenance thereofCulture media therefor
  • A61K 35/407 - LiverHepatocytes
  • A61L 27/38 - Animal cells
  • C12N 5/074 - Adult stem cells
  • A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
  • A61K 35/37 - Digestive system

70.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF HPV-ASSOCIATED DISEASE STATES

      
Application Number 18224632
Status Pending
Filing Date 2023-07-21
First Publication Date 2024-01-25
Owner Children's Hospital Medical Center (USA)
Inventor
  • Wells, Susanne
  • Lange, Taylor
  • Vairamani, Kanimozhi

Abstract

Disclosed are methods and compositions for treating a human papillomavirus (HPV)-related disease or cancer in an individual in need thereof. The method may comprise administering a glucosylceramide synthase (GCS) inhibitor to the individual, for example, miglustat, eligustat, venglustat, T-036, and combinations thereof. The disclosed methods and compositions may be used to treat cancers such as cervical cancer, oropharyngeal cancer, vulvar cancer, anal cancer, penile cancer, vaginal cancer, rectal cancer, squamous cell carcinoma, adenocarcinoma, head and neck cancer, or skin or mucosal lesions such as a common wart, a genital wart, a respiratory wart, a plantar wart, a subungual wart, a periungual wart, a flat wart, and combinations thereof.

IPC Classes  ?

  • A61K 31/437 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
  • C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

71.

System and Method for Automated Risk Assessment for School Violence

      
Application Number 18365493
Status Pending
Filing Date 2023-08-04
First Publication Date 2024-01-18
Owner Children's Hospital Medical Center (USA)
Inventor
  • Barzman, Drew
  • Ni, Yizhao

Abstract

A system and method for predicting risk of violence for an individual (primarily school violence, but not limited to school violence) performs the following steps: (a) receiving responses to questions from an individual; (b) extracting by a computerized annotator words or phrases from the questions and responses; (c) assigning by the annotator extracted word(s) or phrase(s) to at least one of a plurality of pre-defined categories; and (d) automatically identifying and scoring words or phrases that could be classified into the pre-defined categories by a trained machine-learning engine to produce a score reflecting relative risk of violence by the individual. The pre-defined categories include: expression of violent acts or thoughts of the individual; expression of negative feelings, thoughts or acts of others; expression of negative feelings, thoughts or acts of the individual; expression of family discord or tragedies; and expression of protective factors.

IPC Classes  ?

  • G09B 19/00 - Teaching not covered by other main groups of this subclass
  • G09B 7/00 - Electrically-operated teaching apparatus or devices working with questions and answers

72.

BIOMARKER-BASED RISK MODEL TO PREDICT PERSISTENT MULTIPLE ORGAN DYSFUNCTION AFTER CONGENITAL HEART SURGERY

      
Application Number 18325890
Status Pending
Filing Date 2023-05-30
First Publication Date 2024-01-04
Owner Children's Hospital Medical Center (USA)
Inventor Wong, Hector R.

Abstract

Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to persistent multiple organ dysfunction syndrome (MODS) in pediatric patients following cardiopulmonary bypass (CPB). Certain aspects of the disclosure relates to identifying one or more biomarkers associated with septic shock in pediatric patients, obtaining one or more samples from a pediatric patient following CPB, then quantifying from the sample an amount of said biomarkers, wherein the level of said biomarker correlates with a predicted outcome.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

73.

Methods for treating eosinophilic esophagitis

      
Application Number 16281750
Grant Number 11859250
Status In Force
Filing Date 2019-02-21
First Publication Date 2024-01-02
Grant Date 2024-01-02
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Rothenberg, Marc E.
  • Shoda, Tetsuo
  • Wen, Ting

Abstract

The present invention relates to methods for identifying an EoE endotype of a patient and treating the patient with one or more therapies targeted to the patient's disease endotype; and related methods for stratifying patients for clinical trials.

IPC Classes  ?

  • C12Q 1/68 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving nucleic acids
  • C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

74.

REFINED USES OF GABA A RECEPTOR MODULATORS IN TREATMENT OF FRAGILE X SYNDROME

      
Application Number 18252813
Status Pending
Filing Date 2021-11-12
First Publication Date 2023-12-28
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Erickson, Craig A.
  • Gross, Christina
  • Pedapati, Ernest
  • Schmitt, Lauren

Abstract

Identification of Fragile X syndrome (FXS) patients who are likely to respond to a treatment involving a GABAA receptor modulator (e.g., a selective GABAA receptor stimulator), for example, at a low dose, using peripheral Fragile X mental retardation protein (FMRP) as a biomarker, either taken alone or in combination with other biomarkers. Also provided here are methods for treating such FXS patients using the GABAA receptor modulator (e.g., a selective GABAA receptor stimulator at a low dose).

IPC Classes  ?

  • A61K 31/502 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with carbocyclic ring systems, e.g. cinnoline, phthalazine
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 25/00 - Drugs for disorders of the nervous system
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • A61B 5/374 - Detecting the frequency distribution of signals, e.g. detecting delta, theta, alpha, beta or gamma waves

75.

MULTI-CYCLIC IRAK AND FLT3 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number US2023068897
Publication Number 2023/250431
Status In Force
Filing Date 2023-06-22
Publication Date 2023-12-28
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVIC (USA)
  • KUROME THERAPEUTICS, INC. (USA)
Inventor
  • Thomas, Craig, Joseph
  • Hoyt, Scott, Bryan
  • Starczynowski, Daniel, T.
  • Rosenbaum, Jan, Susan

Abstract

Some embodiments of the disclosure include inventive compounds (e.g., compounds of Formula (I)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.

IPC Classes  ?

  • A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
  • A61K 31/4738 - QuinolinesIsoquinolines ortho- or peri-condensed with heterocyclic ring systems
  • A61K 31/435 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
  • A61K 31/4745 - QuinolinesIsoquinolines ortho- or peri-condensed with heterocyclic ring systems condensed with ring systems having nitrogen as a ring hetero atom, e.g. phenanthrolines
  • A61K 31/33 - Heterocyclic compounds

76.

PSEUDOTYPED PARTICLES, MODIFIED CELLS, RELATED COMPOSITIONS, AND RELATED METHODS

      
Application Number 18250996
Status Pending
Filing Date 2021-10-30
First Publication Date 2023-12-21
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Millay, Douglas
  • Hindi, Sajedah

Abstract

Some embodiments of the invention include pseudotyped particles (e.g., pseudo typed exosomes, pseudotyped VSV, and pseudo typed lentiviruses) and modified cells. Other embodiments of the invention include compositions (e.g., pharmaceutical compositions) of pseudotyped particles (e.g., pseudotyped exosomes, pseudotyped VSV, and pseudotyped lentiviruses) and modified cells. Certain embodiments of the invention include methods of making pseudotyped particles (e.g., pseudotyped exosomes, pseudotyped VSV, and pseudotyped lentiviruses) and modified cells. Other embodiments of the invention include methods of administering pseudotyped particles (e.g., pseudotyped exosomes, pseudotyped VSV, and pseudotyped lentiviruses). Further embodiments of the invention include methods of administering pseudotyped particles (e.g., pseudotyped exosomes, pseudotyped VSV, and pseudotyped lentiviruses) to treat diseases (e.g., muscular dystrophy). Additional embodiments of the invention are also discussed herein.

IPC Classes  ?

  • C12N 15/86 - Viral vectors
  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system

77.

MULTI-CYCLIC IRAK AND FLT3 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number US2023068520
Publication Number 2023/245123
Status In Force
Filing Date 2023-06-15
Publication Date 2023-12-21
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES (USA)
  • KUROME THERAPEUTICS, INC. (USA)
Inventor
  • Thomas, Craig, Joseph
  • Hoyt, Scott, Bryan
  • Starczynowski, Daniel, T.
  • Rosenbaum, Jan, Susan

Abstract

Some embodiments of the disclosure include inventive compounds (e.g., compounds of Formula (I)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.

IPC Classes  ?

  • C07D 487/02 - Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups in which the condensed system contains two hetero rings
  • A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems

78.

METHODS AND COMPOSITIONS FOR THE TREATMENT OF CROHN'S DISEASE

      
Application Number 18207707
Status Pending
Filing Date 2023-06-09
First Publication Date 2023-12-14
Owner Children's Hospital Medical Center (USA)
Inventor
  • Minar, Phillip
  • Jegga, Anil G.

Abstract

Disclosed herein anti-TNF therapy companion diagnostics (e.g., a predictive biomarker panel) for management of Crohn's Disease (CD). The disclosed companion diagnostics may be used to identify an appropriate treatment for a patient, and includes, for example, in vitro diagnostic tests or devices that provide information for the use of an anti-TNF therapy. The disclosed methods may be used, in certain aspects, for the identification of patients likely to respond, or as not likely to respond to an anti-TNF agent. The use of the disclosed methods may allow for dose determination, discontinuation, or the administration of combinations of therapeutic agents.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

79.

BIOMARKER-BASED RISK MODEL TO PREDICT DEATH AND PERSISTENT MULTIPLE ORGAN DYSFUNCTION SYNDROME IN PEDIATRIC SEPTIC SHOCK

      
Document Number 03252637
Status Pending
Filing Date 2023-05-31
Open to Public Date 2023-12-07
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Atreya, Mihir
  • Wong, Hector R. (deceased) (décédé(e)/deceased)

IPC Classes  ?

  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing
  • G01N 33/53 - ImmunoassayBiospecific binding assayMaterials therefor
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

80.

HUMANIN, COLIVELIN AND DERIVATIVES FOR THE TREATMENT OF SEPSIS

      
Application Number US2023024265
Publication Number 2023/235549
Status In Force
Filing Date 2023-06-02
Publication Date 2023-12-07
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor Zingarelli, Basilia

Abstract

Disclosed are methods of treating sepsis in an individual in need thereof via administration of a therapeutically effective amount of a humanin protein, or an analog thereof, to the individual. In one aspect, the methods may comprise administration of the humanin analog colivelin for reducing lung, liver and kidney injury and systemic inflammation after an infection.

IPC Classes  ?

  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 31/04 - Antibacterial agents
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

81.

HUMANIN, COLIVELIN AND DERIVATIVES FOR THE TREATMENT OF HEMORRHAGIC SHOCK

      
Application Number US2023024273
Publication Number 2023/235553
Status In Force
Filing Date 2023-06-02
Publication Date 2023-12-07
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor Zingarelli, Basilia

Abstract

Disclosed are methods of treating hemorrhagic shock, for example hemorrhagic shock associated with traumatic injury, gastrointestinal bleeding, spontaneous hemorrhage due to hematologic disorders, uterine hemorrhage, and combinations thereof, in an individual in need thereof. The methods may comprise administering a humanin protein or an analog thereof, for example Humanin G, to the individual.

IPC Classes  ?

  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • A61P 7/04 - AntihaemorrhagicsProcoagulantsHaemostatic agentsAntifibrinolytic agents
  • A61P 9/00 - Drugs for disorders of the cardiovascular system
  • A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

82.

BIOMARKER-BASED RISK MODEL TO PREDICT DEATH AND PERSISTENT MULTIPLE ORGAN DYSFUNCTION SYNDROME IN PEDIATRIC SEPTIC SHOCK

      
Application Number US2023067716
Publication Number 2023/235768
Status In Force
Filing Date 2023-05-31
Publication Date 2023-12-07
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor Atreya, Mihir

Abstract

Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in pediatric patients. Certain aspects of the disclosure relate to identifying one or more biomarkers associated with septic shock in pediatric patients in combination with one or more endothelial-derived biomarkers, obtaining a sample from a pediatric patient having at least one indication of septic shock, then quantifying from the sample an amount of said biomarkers, wherein the level of said biomarker correlates with a predicted outcome.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing
  • G01N 33/53 - ImmunoassayBiospecific binding assayMaterials therefor

83.

METHODS COMPRISING DETECTION OF ADP-HEPTOSE

      
Application Number US2023023313
Publication Number 2023/230108
Status In Force
Filing Date 2023-05-24
Publication Date 2023-11-30
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Starczynowski, Dan
  • Agarwal, Puneet

Abstract

The instant disclosure relates to methods which employ the detection of ADP-D-glycero-β-D- manno-heptose (ADP-heptose) in a biological sample obtained from an individual. In certain aspects, the methods comprise administering a treatment to an individual in which ADP- heptose is detected. The methods may further comprise determining whether the individual has clonal hematopoiesis of indeterminate potential (CHIP) and circulating ADP-heptose.

IPC Classes  ?

  • A61K 31/341 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing
  • A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
  • A61K 31/702 - Oligosaccharides, i.e. having three to five saccharide radicals attached to each other by glycosidic linkages
  • A61K 31/733 - Fructosans, e.g. inulin
  • A61P 35/02 - Antineoplastic agents specific for leukemia
  • C12N 1/00 - Microorganisms, e.g. protozoaCompositions thereofProcesses of propagating, maintaining or preserving microorganisms or compositions thereofProcesses of preparing or isolating a composition containing a microorganismCulture media therefor

84.

PROJECT AUSTIN

      
Serial Number 98290272
Status Registered
Filing Date 2023-11-29
Registration Date 2024-12-31
Owner Children's Hospital & Medical Center, DBA Children's Nebraska ()
NICE Classes  ? 41 - Education, entertainment, sporting and cultural services

Goods & Services

educational services, namely, providing training to emergency service providers in the field of treating and transporting properly medically complex patients

85.

Methods for Treating Eosinophilic Esophagitis

      
Application Number 18229323
Status Pending
Filing Date 2023-08-02
First Publication Date 2023-11-23
Owner Children's Hospital Medical Center (USA)
Inventor
  • Rothenberg, Marc E.
  • Shoda, Tetsuo
  • Wen, Ting

Abstract

The present invention relates to methods for identifying an EoE endotype of a patient and treating the patient with one or more therapies targeted to the patient's disease endotype; and related methods for stratifying patients for clinical trials.

IPC Classes  ?

  • C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

86.

METHODS FOR TREATING ACUTE KIDNEY INJURY

      
Application Number US2023022837
Publication Number 2023/225258
Status In Force
Filing Date 2023-05-19
Publication Date 2023-11-23
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Alder, Matthew
  • Hasson, Denise
  • Goldstein, Stuart
  • Devarajan, Prasad

Abstract

The present invention provides methods relating to the discovery of olfactomedin 4 (OLFM4) as a biomarker for acute kidney injury (AKI) and need for renal replacement therapy, and further as a biomarker for responsiveness to the furosemide stress test (FST). The methods described here are useful in clinical decision support and personalized therapy for AKI, as well as for clinical trial design.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

87.

RAFT CULTURES AND METHODS OF MAKING THEREOF

      
Application Number 18027900
Status Pending
Filing Date 2021-09-22
First Publication Date 2023-11-16
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Shacham-Silverberg, Vered
  • Wells, James Macormack

Abstract

Disclosed herein are esophageal raft culture compositions that more closely resemble native organ structures. These esophageal raft cultures may also be innervated by combination with enteric neural crest cells. These raft culture compositions are advantageous for purposes such as studying organellar function, development, and organization. Also disclosed herein are methods of producing said esophageal raft culture compositions.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

88.

METHODS AND COMPOSITIONS FOR TREATMENT OF IDIOPATHIC PULMONARY FIBROSIS

      
Application Number US2023021978
Publication Number 2023/220332
Status In Force
Filing Date 2023-05-12
Publication Date 2023-11-16
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Madala, Satish, K.
  • Ediga, Harshavardhana
  • Reddy, G, Bhanuprakash

Abstract

Disclosed are compositions and methods for treating idiopathic pulmonary fibrosis (IPF). In one aspect, administration of fibronectin to an individual in need thereof for treatment of IPF is disclosed. Such methods may employ administration of the fibronectin 1 gene or protein, the fibronectin 1 domain gene or protein, or combinations thereof.

IPC Classes  ?

  • A61K 38/16 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
  • A61P 11/00 - Drugs for disorders of the respiratory system

89.

ORGANOID RECOMBINATION

      
Application Number 18029863
Status Pending
Filing Date 2021-09-29
First Publication Date 2023-11-16
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Helmrath, Michael A.
  • Vales, Simon
  • Sundaram, Nambirajan
  • Singh, Akaljot
  • Brown, Nicole

Abstract

Disclosed herein are organoid compositions having heterogeneous combinations of epithelial and mesenchymal components, and methods of making the same by dissociating and recombining the epithelial and mesenchymal components from different sources. These epithelial and mesenchymal components can be derived from the same or different cell type or organoid type. These organoid compositions may exhibit advantageous properties, for example, enhanced in vivo engraftment.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • C12N 5/0775 - Mesenchymal stem cellsAdipose-tissue derived stem cells

90.

Assessing treatment response with estimated number of tumor cells

      
Application Number 17777450
Grant Number 12207939
Status In Force
Filing Date 2020-10-20
First Publication Date 2023-11-09
Grant Date 2025-01-28
Owner
  • University of Cincinnati (USA)
  • Children's Hospital Medical Center (USA)
Inventor
  • Wang, Jinghua
  • He, Lili

Abstract

A method or system for assessing a patient response to a cancer treatment is provided. The method or system includes acquiring at least one base-line radiological image related to a patient immediately before a treatment, acquiring at least one follow-up radiological image during or after the treatment at a predetermined time interval, estimating a first number of specific tumor cells in a region of interest of the patient based on image features of the base-line radiological image using an algorithm or a model, estimating a second number of specific tumor cells in the region of interest of the patient based on image features of the follow-up radiological image using the algorithm or the model, obtaining a difference between the first number of specific tumor cells and the second number of specific tumor cells, and classifying a treatment response to a cancer based on the difference.

IPC Classes  ?

  • A61B 5/00 - Measuring for diagnostic purposes Identification of persons
  • G06T 7/00 - Image analysis
  • G06T 7/45 - Analysis of texture based on statistical description of texture using co-occurrence matrix computation
  • G06T 7/62 - Analysis of geometric attributes of area, perimeter, diameter or volume
  • G06V 10/44 - Local feature extraction by analysis of parts of the pattern, e.g. by detecting edges, contours, loops, corners, strokes or intersectionsConnectivity analysis, e.g. of connected components
  • G06V 10/82 - Arrangements for image or video recognition or understanding using pattern recognition or machine learning using neural networks
  • G06V 20/69 - Microscopic objects, e.g. biological cells or cellular parts

91.

METHODS FOR PREDICTING AND TREATING CHRONIC LUNG ALLOGRAFT DYSFUNCTION

      
Application Number US2023020337
Publication Number 2023/212285
Status In Force
Filing Date 2023-04-28
Publication Date 2023-11-02
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Ziady, Assem
  • Hayes, Don
  • Skala, Emily, J.
  • Siefert, Matthew

Abstract

Disclosed are methods for treating a lung condition selected from one or more of lung allograft dysfunction, rejection, or failure, in an individual who has undergone a lung transplant, comprising a) detecting a biomarker; b) quantifying a biomarker level; and c) comparing the level of a biomarker to a control value; wherein a deviation in a level of biomarker indicates that said individual is likely to develop the lung condition.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

92.

METHODS FOR IMPROVING OUTCOMES FOR HEMATOPOIETIC CELL TRANSPLANT RECIPIENTS AT RISK FOR BRONCHIOLITIS OBLITERANS SYNDROME

      
Application Number US2023020341
Publication Number 2023/212288
Status In Force
Filing Date 2023-04-28
Publication Date 2023-11-02
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Ziady, Assem
  • Myers, Kasiani
  • Wang, Michele
  • Siefert, Matthew
  • Davies, Stella, M.

Abstract

Disclosed are methods for treating an individual at risk for developing bronchiolitis obliterans syndrome (BOS) after hematopoietic stem cell transplant (HSCT), comprising a) detecting a biomarker; b) quantifying a biomarker level; and c) comparing the level of a biomarker to a control value; wherein a deviation in a level of biomarker indicates that said individual is likely to develop the lung condition.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

93.

VASCULARIZED BRAIN ORGANOIDS AND METHODS OF MAKING AND USE

      
Application Number US2023019465
Publication Number 2023/205460
Status In Force
Filing Date 2023-04-21
Publication Date 2023-10-26
Owner CHILDREN’S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Guo, Ziyuan
  • Dao, Lan

Abstract

Disclosed herein are organoid models of the blood-brain barrier and methods for making the same from pluripotent stem cells. These organoids exhibit the complex organization of cells including neurons, endothelial cells, glial cells, and pericytes resembling the natural blood-brain barrier structure. These organoids may be used for studying the functions of the blood-brain barrier and cerebrovascular disorders.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • C12N 5/0793 - Neurons
  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing

94.

COMPOSITIONS AND METHODS COMPRISING R-SPONDINS FOR TREATMENT OF TUMORS

      
Application Number 18007771
Status Pending
Filing Date 2021-06-01
First Publication Date 2023-10-26
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Tang, Yuting
  • Huang, Gang

Abstract

Disclosed herein are methods for treating a cancer in an individual, including administration of an R-spondin protein, for example one or more of R-spondin1, R-spondin2, R-spondin3, R-spondin4, and combinations thereof. Further disclosed are methods of treating an individual having a cancer, the method including determining an R-spondin level in the individual, and administering a treatment to the individual.

IPC Classes  ?

  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • A61P 35/00 - Antineoplastic agents
  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum

95.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF BRONCHIOLITIS OBLITERANS

      
Application Number 18019322
Status Pending
Filing Date 2021-08-05
First Publication Date 2023-10-19
Owner Children's Hospital Medical Center (USA)
Inventor
  • Ziady, Assem
  • Myers, Kasiani
  • Davies, Stella M.
  • Szczesniak, Rhonda
  • Gecili, Emrah
  • Skala, Emily J.
  • Mousa, Nadeem Ayoub Ali
  • Towe, Christopher
  • Walkup, Laura
  • Woods, Jason

Abstract

Disclosed herein are compositions and methods for the treatment of bronchiolitis obliterans syndrome (BOS), in particular, in association with hematopoietic stem cell transplant (HSCT recipients, and methods for patients for effective treatment of same using protein and functional biomarkers. In certain aspect, the disclosed compositions and methods may be used for early identification of individuals likely to develop BOS such that optimal treatment may be provided, wherein the methods may employ detection and assessment of one or both of a biomarker and lung function.

IPC Classes  ?

  • A61K 31/7052 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
  • A61K 31/18 - Sulfonamides
  • A61K 31/40 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
  • A61K 31/22 - Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
  • A61P 11/00 - Drugs for disorders of the respiratory system
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

96.

METHODS AND COMPOSITIONS RELATING TO PERSONALIZED PAIN MANAGEMENT

      
Application Number US2023017011
Publication Number 2023/196169
Status In Force
Filing Date 2023-03-31
Publication Date 2023-10-12
Owner CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Chidambaran, Vidya
  • Ji, Hong
  • Martin, Lisa J.

Abstract

The disclosure relates to methods for pain management in the perioperative context, particularly through the use of one or more biomarkers such as the DNA methylation status in genes of the PARK16 locus.

IPC Classes  ?

  • A61K 31/245 - Amino benzoic acid types, e.g. procaine, novocaine
  • A61K 31/706 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
  • A61K 31/7068 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
  • A61P 23/00 - Anaesthetics

97.

MULTI-CYCLIC IRAK AND FLT3 INHIBITING COMPOUNDS AND USES THEREOF

      
Application Number 18018742
Status Pending
Filing Date 2021-07-31
First Publication Date 2023-09-28
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN (USA)
  • KUROME THERAPEUTICS, INC. (USA)
Inventor
  • Hoyt, Scott Bryan
  • Thomas, Craig Joseph
  • Finocchio, Chris James
  • Starczynowski, Daniel T.
  • Tawa, Gregory J.
  • Gracia Maldonado, Gabriel
  • Rosebaum, Jan Susan

Abstract

Some embodiments of the disclosure include disclosed compounds (e.g., compounds of Formula (I)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the disclosed compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the disclosed IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.

IPC Classes  ?

  • C07D 471/04 - Ortho-condensed systems
  • C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca

98.

DELIVERY METHODS USING PLATELETS

      
Application Number US2023064831
Publication Number 2023/183855
Status In Force
Filing Date 2023-03-22
Publication Date 2023-09-28
Owner CHILDREN’S HOSPITAL MEDICAL CENTER (USA)
Inventor
  • Takebe, Takanori
  • Kimura, Masaki
  • Dunn, Andrew
  • Iguchi, Takuma

Abstract

Disclosed herein are methods of transferring exogenous material using platelets. The methods comprise administering the platelets with the exogenous material to a recipient. The platelets may be obtained by directly contacting the platelets, or contacting megakaryocytes that produce the platelets.

IPC Classes  ?

99.

IDENTIFICATION OF SURGERY CANDIDATES USING NATURAL LANGUAGE PROCESSING

      
Application Number 18123890
Status Pending
Filing Date 2023-03-20
First Publication Date 2023-09-21
Owner
  • Children's Hospital Medical Center (USA)
  • The Regents of the University of Colorado (USA)
Inventor
  • Pestian, John P.
  • Glauser, Tracy A.
  • Holland, Katherine D.
  • Standridge, Shannon Michelle
  • Greiner, Hansel M.
  • Cohen, Kevin Bretonnel

Abstract

The present invention relates to computer-based clinical decision support tools including, computer-implemented methods, computer systems, and computer program products for clinical decision support. These tools assist the clinician in identifying epilepsy patients who are candidates for surgery and utilize a combination of natural language processing, corpus linguistics, and machine learning techniques.

IPC Classes  ?

  • G06F 40/20 - Natural language analysis
  • G06Q 10/10 - Office automationTime management
  • G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
  • G06F 40/40 - Processing or translation of natural language
  • G16H 10/60 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for patient-specific data, e.g. for electronic patient records
  • G16H 20/40 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to mechanical, radiation or invasive therapies, e.g. surgery, laser therapy, dialysis or acupuncture
  • G16Z 99/00 - Subject matter not provided for in other main groups of this subclass

100.

METHODS AND COMPOSITIONS FOR TREATMENT OF FRAGILE X SYNDROME

      
Application Number 18005757
Status Pending
Filing Date 2021-07-16
First Publication Date 2023-09-21
Owner
  • CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
  • FORGE BIOLOGICS, INC. (USA)
Inventor
  • Pedapati, Ernest
  • Gross, Christina
  • Erickson, Craig
  • Dismuke, David
  • De Silva, Erandi Kanchana

Abstract

Methods for alleviating symptoms in a Fragile X Syndrome (FXS) patient using adeno-associated viral (AAV) 9 viral particles encoding a wild-type human fragile X mental retardation 1 (FMR1) protein (human FMRP). Also provided herein are methods to determine suitable doses of AAV9 viral particles for a FXS patient to alleviate at least one symptom associated with FXS, as well as methods for monitoring treatment efficacy.

IPC Classes  ?

  • C12N 15/86 - Viral vectors
  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61P 43/00 - Drugs for specific purposes, not provided for in groups
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