The present disclosure provides, among other things, a method of isolating and expanding gamma delta (γδ) T cells, wherein the method comprises: (a) isolating non- hematopoeitic tissue by biopsy or explant, and (b) culturing the isolated non-hematopoeitic tissue in the absence of a three-dimensional scaffold or grid, thereby expanding and isolating gamma delta (γδ) T cells.
Improving the solubility of an organic compound. A cocrystal of (1) 6-ethyl-N-[1-(hydroxyacetyl)piperidin-4-yl]-1-methyl-4-oxo-5-(2-oxo-2-phenylethyl)-3-(2,2,2-trifluoroethoxy)-4,5-dihydro-1H-pyrrolo[3,2-c]pyridine-2-carboxamide and (2) L-malic acid or L-tartaric acid.
The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A.
Disclosed herein are improved methods and compositions for diagnosing, preventing and/or treating Huntington's Disease. Among other things, provided herein is a gene therapy construct encoding a non-naturally occurring codon-optimized transcription factor (ZFP-TF) comprising a zinc-finger protein (ZFP) sequence and a sequence encoding a transcriptional repression domain, wherein the ZFP-TF expression is driven by a phosphoglycerate kinase 1 (PGK), ubiquitin C (UBC), an EFS, or an EF1alpha promoter.
A61K 38/00 - Préparations médicinales contenant des peptides
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
A61P 25/14 - Médicaments pour le traitement des troubles du système nerveux pour traiter les mouvements anormaux, p.ex. chorée, dyskinésie
C07K 14/47 - Peptides ayant plus de 20 amino-acides; Gastrines; Somatostatines; Mélanotropines; Leurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
C12N 9/42 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons bêta-glucosidiques-1, 4, p.ex. cellulase
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
A61P 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteurs; Vecteurs; Utilisation d'hôtes pour ceux-ci; Régulation de l'expression
The invention relates to novel compositions and methods of using maribavir which enhance its effectiveness in medical therapy, as well as to maribavir isomers and methods of use thereof for counteracting the potentially adverse effects of maribavir isomerization in vivo in the event it occurs.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p.ex. composés antiphlogistiques et pour le cœur
G01N 33/94 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir des narcotiques
G09B 19/00 - Enseignement non couvert par d'autres groupes principaux de la présente sous-classe
Disclosed is a method for predicting the gene transfer rate in animal cell and/or the number of gene-transferred cells in the animal cells, the method comprising (1) a step for measuring a component in a supernatant of a culture obtained by the culturing of gene-transferred animal cells and (2) a step for predicting the gene transfer rate and/or the number of gene-transferred cells on the basis of a value measured in step (1).
C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismes; Compositions à cet effet; Procédés pour préparer ces compositions faisant intervenir des acides nucléiques
G01N 33/50 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique
8.
BIOMARKERS AND METHODS FOR TREATMENT WITH NAE INHIBITORS
Disclosed herein are markers whose mutational status is associated with sensitivity to treatment with NAE inhibitors. Mutational status is determined by measurement of characteristics of markers corresponding to the marker genes. Compositions and methods are provided to assess markers of marker genes to predict response to NAE inhibition treatment.
A61K 31/519 - Pyrimidines; Pyrimidines hydrogénées, p.ex. triméthoprime condensées en ortho ou en péri avec des hétérocycles
A61K 31/496 - Pipérazines non condensées contenant d'autres hétérocycles, p.ex. rifampine, thiothixène
A61K 31/706 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à six chaînons avec l'azote comme hétéro-atome d'un cycle
A61P 35/02 - Agents anticancéreux spécifiques pour le traitement de la leucémie
A61P 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes
9.
METHODS AND COMPOSITIONS FOR TREATING ULCERATIVE COLITIS
The invention provides methods and compositions related to methods for determining whether a human subject having ulcerative colitis will respond to treatment with a humanized antibody having binding specificity for human α4β7 integrin for treating ulcerative colitis. The invention provides response markers that can be used to identify responders of ulcerative colitis.
G01N 33/68 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir des protéines, peptides ou amino-acides
Methods of treating at least one cognitive symptom, e.g., at least one cognitive symptom associated with schizophrenia, comprising administering at least one compound chosen from Compound (I) and pharmaceutically acceptable salts thereof are disclosed. Also disclosed are methods of increasing D-serine levels, synaptic plasticity, and/or long-term potentiation comprising administering at least one compound chosen from Compound (I) and pharmaceutically acceptable salts thereof.
Methods of treating at least one cognitive symptom, e.g., at least one cognitive symptom associated with schizophrenia, comprising administering at least one compound chosen from Compound (I) and pharmaceutically acceptable salts thereof are disclosed. Also disclosed are methods of increasing D-serine levels, synaptic plasticity, and/or long-term potentiation comprising administering at least one compound chosen from Compound (I) and pharmaceutically acceptable salts thereof.
A61K 9/28 - Dragées; Pilules ou comprimés avec revêtements
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p.ex. composés antiphlogistiques et pour le cœur
A61P 25/18 - Antipsychotiques, c. à. d. neuroleptiques; Médicaments pour le traitement de la manie ou de la schizophrénie
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p.ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
Disclosed is a method for producing regulatory T cells, the method comprising:
(1) differentiating cells that can differentiate into regulatory T cells, into which an expression construct is introduced, into regulatory T cells, the expression construct comprising:
(a) conserved non-coding sequence (CNS) 1, CNS2, and CNS3 of Foxp3 gene;
(b) a promoter; and
(c) a nucleic acid encoding FOXP3.
Also disclosed are regulatory T cells obtained by the method, and a pharmaceutical composition comprising the regulatory T cells.
The present disclosure provides novel methods and compositions for using maribavir in the treatment of pediatric and adolescent patient populations. The present disclosure also provides compositions of maribavir in the form of a powder for oral suspension, which are useful for patients with difficulty swallowing pills, including pediatric and adolescent patient populations.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61P 31/22 - Antiviraux pour le traitement des virus ADN des virus de l'herpès
13.
USE OF AN OREXIN 2 RECEPTOR AGONIST FOR IMPROVING RESPIRATORY FUNCTION DURING SLEEP
Disclosed are uses of an orexin type 2 receptor (OX2R) agonist at non-awakening concentrations to treat sleep apnea. Also disclosed are compositions and methods for administering an OX2R agonist to a subject (e.g., mammal) in need thereof at a dose that provides a blood plasma concentration of the OX2R agonist that is at or below the maximum non-awakening blood plasma concentration of the OX2R agonist. Also disclosed are compositions and methods for treating sleep apnea in a subject in need thereof by maintaining the blood plasma concentration of an OX2R agonist following administration which is at or below the maximum non-awakening concentration of the OX2R agonist.
A61K 31/40 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p.ex. guanéthidine ou rifamycines ayant des cycles à cinq chaînons avec un azote comme seul hétéro-atome d'un cycle, p.ex. sulpiride, succinimide, tolmétine, buflomédil
A61K 31/445 - Pipéridines non condensées, p.ex. pipérocaïne
A61K 45/00 - Préparations médicinales contenant des ingrédients actifs non prévus dans les groupes
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
Disclosed are: a method for producing a cell population comprising T precursor cells, the method comprising step (1) for culturing a cell population containing CD34+ cells in the presence of an aromatic hydrocarbon receptor antagonist and an LSD1 degradation inducing agent; a cell population comprising T precursor cells obtained by this method; a medicament containing the cell population comprising T precursor cells; and a T cell progenitor inducing agent comprising an aromatic hydrocarbon receptor antagonist and an LSD1 degradation inducer.
The present invention provides, among other things, improved methods for purifying I2S protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant I2S protein can be purified from unprocessed biological materials, such as, I2S-containing cell culture medium, using a process involving as few as four chromatography columns.
The present disclosure provides novel methods and compositions for using maribavir in the treatment of pediatric and adolescent patient populations. The present disclosure also provides compositions of maribavir in the form of a powder for oral suspension, which are useful for patients with difficulty swallowing pills, including pediatric and adolescent patient populations.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
Disclosed is a method for measuring a cell concentration in a sample, the method including the following steps of: (1) measuring the number of copies of a specific gene and a gene of an external standard in a plurality of standard samples each having a known cell concentration by using digital PCR (dPCR); (2) normalizing the measured value of the specific gene measured in the step (1) using the measured value of the external standard and creating a calibration curve on the basis of the normalized value and the known cell concentration; (3) measuring the number of copies of the same specific gene and gene of the external standard in a sample having an unknown cell concentration by using dPCR; and (4) normalizing the measured value of the specific gene measured in the step (3) using the measured value of the external standard and determining a cell concentration using the calibration curve created in the step (2) from the normalized value.
The present invention assists appropriate information input with respect to the monitoring of Parkinson's disease patients. This information terminal comprises: a screen control unit configured so that in a state where a video list screen that includes a list of videos capturing a Parkinson's disease patient is displayed, the screen control unit displays, if any of the videos is selected, a condition list screen that includes a list of condition information items that represents conditions of the Parkinson's disease patient; an addition unit configured so that in a state where the condition list screen is displayed, and if condition information corresponding to a condition of the Parkinson's disease patient when the selected video was taken is selected, the addition unit adds the selected condition information to the selected video; and a transmission unit that transmits the selected video to which the selected condition information has been added to a server device that can be accessed by a healthcare worker examining the Parkinson's disease patient or by a caregiver providing care to the Parkinson's disease patient.
G16H 80/00 - TIC spécialement adaptées pour faciliter la communication entre les professionnels de la santé ou les patients, p.ex. pour le diagnostic collaboratif, la thérapie collaborative ou la surveillance collaborative de l’état de santé
G16H 10/00 - TIC spécialement adaptées au maniement ou au traitement des données médicales ou de soins de santé relatives aux patients
G16H 30/40 - TIC spécialement adaptées au maniement ou au traitement d’images médicales pour le traitement d’images médicales, p.ex. l’édition
The present invention provides, among other things, improved methods for purifying arylsulfatase A (ASA) protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant ASA protein can be purified from unprocessed biological materials, such as, ASA-containing cell culture medium, using a process involving as few as four chromatography columns and only one step of post-chromatographic ultrafiltration/diafiltration.
The present invention aims to provide a novel drug delivery system (DDS) technique capable of selectively delivering a drug (compound containing oligonucleotides for producing at least partially functional dystrophin protein) to muscle tissues such as cardiac muscle, skeletal muscle and the like and efficiently introducing the drug into the muscle cells. The present invention relates to a conjugate or a salt thereof including the following: (1) a peptide that binds to a transferrin receptor, and contains the amino acid sequence shown in SEQ ID NO: 1 (Ala-Val-Phe-Val-Trp-Asn-Tyr-Tyr-Ile-Ile-Ser-Cys); or an amino acid sequence resulting from substitution, deletion, addition, and/or insertion of not less than one and not more than 10 amino acid residues in the amino acid sequence shown in SEQ ID NO: 1, and (2) a compound comprising an oligonucleotide for producing an at least partially functional dystrophin protein.
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c. à d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
A61K 38/17 - Peptides ayant plus de 20 amino-acides; Gastrines; Somatostatines; Mélanotropines; Leurs dérivés provenant d'humains
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p.ex. oligonucléotides anti-sens
21.
FACILITATED DELIVERY OF CONCENTRATED ANTIBODY FORMULATIONS USING HYALURONIDASE
Provided is a concentrated pharmaceutical formulation of an immune globulin (IG), and convenient methods for the subcutaneous administration of this pharmaceutical formulation in a warmed state. Such products can be used in methods of treating IG-treatable diseases or conditions. Also provided are combinations, compositions and kits containing an immune globulin (IG) composition and a soluble hyaluronidase composition formulated for subcutaneous administration.
Disclosed herein are computer-implemented method, system, and computer-program product (computer-readable storage medium) embodiments of image scoring for intestinal pathology. An embodiment includes receiving, via at least one processor, an output of an imaging device. The output of the imaging device may include a plurality of image frames forming at least a subset of a set of image frames depicting an inside surface of a digestive organ of a given patient; and decomposing, via at least one machine learning (ML) algorithm, at least one image frame of the plurality of image frames into a plurality of regions of interest. The at least one region of interest may be defined by determining that an edge value exceeds a predetermined threshold. At least one processor may automatically assign a first score based at least in part on the edge value for each region of interest and automatically shuffle the set of image frames.
G06F 16/383 - Recherche caractérisée par l’utilisation de métadonnées, p.ex. de métadonnées ne provenant pas du contenu ou de métadonnées générées manuellement utilisant des métadonnées provenant automatiquement du contenu
G06F 18/21 - Conception ou mise en place de systèmes ou de techniques; Extraction de caractéristiques dans l'espace des caractéristiques; Séparation aveugle de sources
G06V 10/764 - Dispositions pour la reconnaissance ou la compréhension d’images ou de vidéos utilisant la reconnaissance de formes ou l’apprentissage automatique utilisant la classification, p.ex. des objets vidéo
G06V 10/82 - Dispositions pour la reconnaissance ou la compréhension d’images ou de vidéos utilisant la reconnaissance de formes ou l’apprentissage automatique utilisant les réseaux neuronaux
23.
AAV8 CAPSID VARIANTS WITH ENHANCED LIVER TARGETING
The present disclosure provides variant AAV8 capsids that exhibit altered capsid properties, e.g., improved transduction efficiency and/or specificity for the liver. The present disclosure further provides nucleic acids encoding the variant AAV8 capsids, recombinant AAV (rAAV) vectors comprising the variant AAV8 capsids, as well as host cells and compositions comprising the same. The present disclosure further provides methods of delivering a gene product to a subject and methods of treatment of a liver-borne blood disorder, the methods generally involving administering an effective amount of the rAAV vectors to a subject in need thereof.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acides; Gastrines; Somatostatines; Mélanotropines; Leurs dérivés provenant de virus
The present disclosure provides methods for identifying individuals at risk for developing Alpha- 1 Antitrypsin Deficiency-associated liver disease (AATD-LD), diagnosing individuals with AATD-LD, and administering therapeutic agents to individuals having or predisposed to AATD-LD.
G01N 33/68 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir des protéines, peptides ou amino-acides
The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.
A61K 31/506 - Pyrimidines; Pyrimidines hydrogénées, p.ex. triméthoprime non condensées et contenant d'autres hétérocycles
C07D 519/00 - Composés hétérocycliques contenant plusieurs systèmes de plusieurs hétérocycles déterminants condensés entre eux ou condensés avec un système carbocyclique commun non prévus dans les groupes ou
26.
COMPOSITIONS AND METHODS FOR DELIVERY OF NUCLEIC ACIDS TO CELLS
The disclosure features novel lipid nanoparticle formulations and use thereof. Lipid nanoparticles include cationic ionizable lipids, helper lipids, sterols, and conjugated lipids. Lipid nanoparticles further including therapeutics and/or prophylactics such as DNA are useful in the delivery of therapeutics and/or prophylactics to cells or organs (e.g., mammalian cells or organs) to, for example, regulate polypeptide, protein, or gene expression.
The present disclosure relates to sequences and methods for delivery of DNA and RNA using, for example, DNA nuclear targeting sequences (DTSs). These sequences provide more efficient delivery of target DNA and RNA into cells. These sequences are also particularly effective for targeting liver cells, for example, for treatment of inherited liver and systemic disorders, as well as acquired genetic disorders, and also serve as a treatment method for numerous diseases and disorders.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
C12N 15/11 - Fragments d'ADN ou d'ARN; Leurs formes modifiées
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
28.
PET TRACER COMPOUNDS FOR TRACING ALPHA-GALACTOSIDASE A AND REACTION INTERMEDIATES, AND METHODS OF USE
C12Q 1/34 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismes; Compositions à cet effet; Procédés pour préparer ces compositions faisant intervenir une hydrolase
C07B 59/00 - Introduction d'isotopes d'éléments dans les composés organiques
Disclosed herein are engineered immune cells that specifically recognizes mesothelin and expresses IL-15 and optionally CCL19. Also disclosed herein are isolated nucleic acid molecules comprising a polynucleotide encoding a chimeric antigen receptor (CAR) comprising an antibody that specifically recognizes mesothelin, and a 4-IBB intracellular region; and a polynucleotide encoding IL-15; and optionally a polynucleotide encoding CCL19, vectors, pharmaceutical compositions comprising the nucleic acid molecules, and methods of using the engineered immune cells.
C07K 16/30 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire provenant de cellules de tumeurs
C12N 5/0783 - Cellules T; Cellules NK; Progéniteurs de cellules T ou NK
30.
VEDOLIZUMAB FOR THE TREATMENT OF FISTULIZING CROHN'S DISEASE
A method for treating a human patient suffering from fistulizing Crohn's disease, comprising administering to a patient suffering from fistulizing Crohn's disease, a humanized antibody having binding specificity for human α4β7 integrin, wherein the human patient has a seton that was surgically placed prior to administration of the antibody, and wherein the dosing regimen induces fistula (e) healing.
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
G01N 33/00 - Recherche ou analyse des matériaux par des méthodes spécifiques non couvertes par les groupes
G01N 33/564 - Tests immunologiques; Tests faisant intervenir la formation de liaisons biospécifiques; Matériaux à cet effet pour complexes immunologiques préexistants ou maladies auto-immunes
G01N 33/68 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir des protéines, peptides ou amino-acides
C12N 15/52 - Gènes codant pour des enzymes ou des proenzymes
A61K 38/47 - Hydrolases (3) agissant sur des composés glycosyliques (3.2), p.ex. cellulases, lactases
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
C12N 9/26 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons alpha-glucosidiques-1, 4, p.ex. hyaluronidase, invertase, amylase
The present invention provides, among other things, methods of engineering a re- editable target locus in a cell, comprising inserting an exogenous re-editable template sequence to a target locus and methods of engineering a re-editable cell comprising a re- editable template sequence flanked by two homology arms such that the re-editable template sequence is inserted into a target locus by homologous recombination, and methods of using the same. In some aspects, provided is a re-editable template sequence, wherein the template sequence has no substantial sequence similarity to any region in the genome, and wherein the template sequence is recognizable by a genome editing system.
C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
C12N 15/11 - Fragments d'ADN ou d'ARN; Leurs formes modifiées
33.
SOLID STATE FORMS OF HYDROCHLORIDE SALT OF ((1S,2S,4R)-4-{4-[(1S)-2,3-DIHYDRO-1H-INDEN-1-YLAMINO]-7H-PYRROLO[2,3-D]PYRIMIDIN-7-YL}-2-HYDROXYCYCLOPENTYL)METHYL SULFAMATE
Disclosed herein are solid state forms of ((1S,2S,4R)-4-{4-[(1S)-2,3-dihydro-1H-inden-1-ylamino]-7H-pyrrolo[2,3-d]pyrimidin-7-yl}-2-hydroxycyclopentyl)methyl sulfamate hydrochloride, compositions thereof, methods for their preparation, and methods for their use.
The present disclosure discloses compositions comprising maribavir, methods of providing the same, and compositions providing intermediates useful in providing maribavir.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 31/4184 - 1,3-Diazoles condensés avec des carbocycles, p.ex. benzimidazoles
The present disclosure discloses compositions comprising maribavir, methods of providing the same, and compositions providing intermediates useful in providing maribavir.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 31/4184 - 1,3-Diazoles condensés avec des carbocycles, p.ex. benzimidazoles
The present disclosure discloses compositions comprising maribavir, methods of providing the same, and compositions providing intermediates useful in providing maribavir.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 31/4184 - 1,3-Diazoles condensés avec des carbocycles, p.ex. benzimidazoles
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
wherein each symbol is as described in the specification, or a salt thereof has an orexin type 2 receptor agonist activity, and is useful as an agent for the prophylaxis or treatment of narcolepsy.
C07D 207/14 - Atomes d'azote ne faisant pas partie d'un radical nitro
C07D 403/06 - Composés hétérocycliques contenant plusieurs hétérocycles, comportant des atomes d'azote comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant deux hétérocycles liés par une chaîne carbonée ne contenant que des atomes de carbone aliphatiques
C07D 407/08 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'oxygène comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant deux hétérocycles liés par une chaîne carbonée contenant des cycles alicycliques
C07D 417/06 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre et d'azote comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant deux hétérocycles liés par une chaîne carbonée contenant uniquement des atomes de carbone aliphatiques
Provided is a compound represented by the formula (I):
Provided is a compound represented by the formula (I):
Provided is a compound represented by the formula (I):
wherein each symbol is as defined in the specification, or a salt thereof, which has an AMPA (α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid) receptor potentiating action. The compound of the present invention is useful as a prophylactic or therapeutic drug for depression, schizophrenia, Alzheimer's disease or attention deficit hyperactivity disorder (ADHD) and the like.
The present disclosure relates to methods of expanding or producing CD56+/CD3-cells comprising contacting the CD56+/CD3- cells with an NKp46 agonist and/or a CD30 agonist.
Methods for treating prostate cancer, including advanced prostate cancer, in a subject in need thereof, include administering once-daily to the subject, at least 80 mg of N-(4-(1-(2,6-difluorobenzyl)-5-((dimethylamino)methyl)-3-(6-methoxy-3-pyridazinyl)-2,4-dioxo-1,2,3,4-tetrahydrothieno[2,3-d]pyrimidin-6-yl)phenyl)-N′-methoxyurea, or a corresponding amount of a pharmaceutically acceptable salt thereof. Another method includes: administering once-daily to the subject in need thereof, an oral load dose formulation having from 240 mg to 480 mg of N-(4-(1-(2,6-difluorobenzyl)-5-((dimethylamino)methyl)-3-(6-methoxy-3-pyridazinyl)-2,4-dioxo-1,2,3,4-tetrahydrothieno[2,3-d]pyrimidin-6-yl)phenyl)-N′-methoxyurea, or a corresponding amount of a pharmaceutically acceptable salt thereof, and thereafter administering once-daily to the subject, an oral maintenance dose formulation having 80 mg to 160 mg of N-(4-(1-(2,6-difluorobenzyl)-5-((dimethylamino)methyl)-3-(6-methoxy-3-pyridazinyl)-2,4-dioxo-1,2,3,4-tetrahydrothieno[2,3-d]pyrimidin-6-yl)phenyl)-N′-methoxyurea, or a corresponding amount of a pharmaceutically acceptable salt thereof.
A61K 31/517 - Pyrimidines; Pyrimidines hydrogénées, p.ex. triméthoprime condensées en ortho ou en péri avec des systèmes carbocycliques, p.ex. quinazoline, périmidine
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
Disclosed herein are isolated nucleic acid molecules comprising a polynucleotide encoding a chimeric antigen receptor (CAR) comprising an antibody that specifically recognizes human mesothelin, a CD8 hinge region, a CD8 transmembrane region, a 4-1BB intracellular region and a CD3ζ intracellular region; a polynucleotide encoding IL-7; and a polynucleotide encoding CCL19. Also disclosed herein include vectors, modified immune cells, and pharmaceutical compositions comprising the nucleic acid molecules and methods of use.
C07K 16/30 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire provenant de cellules de tumeurs
C12N 5/0783 - Cellules T; Cellules NK; Progéniteurs de cellules T ou NK
43.
LIBRARIES OF GENETIC PACKAGES COMPRISING NOVEL HC CDR3 DESIGNS
Provided are compositions and methods for preparing and identifying antibodies having CDR3s that vary in sequence and in length from very short to very long which in certain embodiments may bind to a carbohydrate moiety or the active site of an enzyme. Libraries coding for antibodies with the CDR3s are also provided. The libraries can be provided by modifying a pre-existing nucleic acid library.
C40B 50/06 - Procédés biochimiques, p.ex. utilisant des enzymes ou des micro-organismes viables entiers
C07K 16/00 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux
C07K 16/12 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant de bactéries
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
C40B 40/08 - Bibliothèques comprenant de l'ARN ou de l'ADN codant des protéines, p.ex. bibliothèques de gènes
The present application provides BCMA targeting chimeric antigen receptor (CAR) comprising a BCMA binding region and an intracellular costimulatory domain derived from DAP10. Further provided are engineered immune effector cells (such as NK cells) comprising the chimeric antigen receptors. Pharmaceutical compositions, kits and methods of treating cancer are also provided.
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
G01N 33/86 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir le temps de coagulation du sang
46.
COMPOSITIONS TARGETING BCMA AND METHODS OF USE THEREOF
This present invention relates to BCMA binders (e.g. antibodies) and chimeric antigen receptor (CAR) constructs comprising a BCMA antigen binding molecule. The BCMA binders specifically bind to BCMA. The present BCMA CARs further comprise a hinge region (e.g., CD28 hinge), a transmembrane domain, and one or more intracellular NK cell signalling domains. NK cells expressing a BCMA CAR has increased efficacy in killing cancer cells. Provided herein also include therapeutic uses of the BCMA binders and BCMA CARs.
A61K 35/17 - Lymphocytes; Lymphocytes B; Lymphocytes T; Cellules tueuses naturelles; Lymphocytes activés par un interféron ou une cytokine
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61K 47/20 - Composés organiques, p.ex. hydrocarbures naturels ou synthétiques, polyoléfines, huile minérale, gelée de pétrole ou ozocérite contenant du soufre, p.ex. sulfoxyde de diméthyle [DMSO], docusate, laurylsulfate de sodium ou acides aminosulfoniques
A61K 47/26 - Hydrates de carbone, p.ex. polyols ou sucres alcoolisés, sucres aminés, acides nucléiques, mono-, di- ou oligosaccharides; Leurs dérivés, p.ex. polysorbates, esters d’acide gras de sorbitan ou glycyrrhizine
A61K 47/42 - Protéines; Polypeptides; Leurs produits de dégradation; Leurs dérivés p.ex. albumine, gélatine ou zéine
A61P 35/02 - Agents anticancéreux spécifiques pour le traitement de la leucémie
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM (USA)
TAKEDA PHARMACEUTICAL COMPANY LIMITED (Japon)
Inventeur(s)
Rezvani, Katy
Basar, Rafet
Lin, Paul
Curley, Michael David
Talarico, Leeann
Vishwanath, Prashanth
Meador, James Wilson, Iii
Abrégé
The present application provides BCMA targeting chimeric antigen receptor (CAR) comprising a BCMA binding region and an intracellular costimulatory domain derived from DAP10. Further provided are engineered immune effector cells (such as NK cells) comprising the chimeric antigen receptors. Pharmaceutical compositions, kits and methods of treating cancer are also provided.
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
The present invention provides a heterocyclic compound having a HDAC6 inhibitory action, which is useful for the treatment of central nervous system diseases including neurodegenerative diseases, and the like, and a medicament comprising the compound. The present invention relates to a compound represented by the formula (I):
The present invention provides a heterocyclic compound having a HDAC6 inhibitory action, which is useful for the treatment of central nervous system diseases including neurodegenerative diseases, and the like, and a medicament comprising the compound. The present invention relates to a compound represented by the formula (I):
The present invention provides a heterocyclic compound having a HDAC6 inhibitory action, which is useful for the treatment of central nervous system diseases including neurodegenerative diseases, and the like, and a medicament comprising the compound. The present invention relates to a compound represented by the formula (I):
wherein each symbol is as defined in the description, or a salt thereof.
C07D 413/14 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle contenant au moins trois hétérocycles
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p.ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C07D 413/10 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle contenant deux hétérocycles liés par une chaîne carbonée contenant des cycles aromatiques
49.
PYRAZOLOPYRIMIDINE DERIVATIVES AS INHIBITORS OF NLRP3
The invention provides a compound of Formula (I), or a pharmaceutically acceptable salt thereof: wherein L, X, Y, R1, R2, R3, R4, R5, R6, R7and R8 are as defined in the specification, for treatment of a disease, disorder or condition associated with NLRP3, including a disease, disorder or condition associated with a heterozygous gain of function mutation in the NLRP3 gene such as cryopyrin-associated periodic syndrome (CAPS).
An antibody-resin coupling apparatus quickly and efficiently activates resin beads and couples them to antibodies, while preventing breakdown and crosslinking of the beads, thereby improving downstream column purification processes, extending the usable life of the resin beads, and increasing molecule capture efficiency of the resultant resin-antibody complexes, to allow improved isolation and purification of factor VIII molecules or other drug compounds.
B01J 19/18 - Réacteurs fixes avec éléments internes mobiles
B01D 29/01 - Filtres à éléments filtrants stationnaires pendant la filtration, p.ex. filtres à aspiration ou à pression, non couverts par les groupes ; Leurs éléments filtrants avec des éléments filtrants plats
B01F 27/13 - Agitateurs à cadre ajouré ou à cage non prévus dans d’autres groupes de la présente sous-classe
B01F 35/45 - Fermetures ou portes spécialement adaptées aux récipients de mélange; Mécanismes de fonctionnement à cet effet
The present disclosure provides methods, pharmaceutical compositions, and kits for treating cancer in patients in need thereof. The methods comprise administering to a patient in need a STING (stimulator of interferon genes) agonist, such as Compound No. 14 as defined in the description, or a pharmaceutically acceptable salt thereof, in combination with one or more checkpoint inhibitors and radiation. Also provided are medicaments for use in treating cancer.
The invention relates to novel compositions and methods of using maribavir which enhance its effectiveness in medical therapy, as well as to maribavir isomers and methods of use thereof for counteracting the potentially adverse effects of maribavir isomerization in vivo in the event it occurs.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p.ex. composés antiphlogistiques et pour le cœur
G01N 33/94 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir des narcotiques
G09B 19/00 - Enseignement non couvert par d'autres groupes principaux de la présente sous-classe
This disclosure features novel lipid nanoparticle formulations and uses thereof. The lipid nanoparticle ("LNP") includes an encapsulated therapeutic agent and an aqueous solution comprising a salt and an anionic polymer, wherein the salt and the anionic polymer are dissolved in the aqueous solution, thereby forming polymer coated lipid nanoparticle ("PCLNP"). Lipid nanoparticles of this disclosure are useful in the process of lyophilization or freeze drying and decrease nanoparticle aggregation and maintain efficacy once reconstituted.
A61K 47/32 - Composés macromoléculaires obtenus par des réactions faisant intervenir uniquement des liaisons non saturées carbone-carbone, p.ex. carbomères
54.
T CELLS FOR USE IN TREATING RELAPSED OR REFRACTORY ACUTE MYELOID LEUKEMIA
The present disclosure provides, among other things, methods for treating relapsed or refractory acute myeloid leukemia by administering to a subject in need thereof a therapeutically effective amount of an allogeneic composition comprising VDelta1+ (Vδ1+) gamma delta (γδ) T cells such that one or more symptoms or biomarkers is improved after treatment. The present disclosure also provides suitable doses of compositions comprising allogeneic Vδ1+gamma delta (γδ) T cells for administration to a subject suffering from relapsed or refractory AML. In some embodiments, the Vδ1+gamma delta (γδ) T cells are untransduced.
The invention provides a complex for delivering an active substance, comprising a delivery vehicle for the active substance and a ligand specific to a target cell, the ligand being added to the outer surface of the delivery vehicle, wherein the delivery vehicle is comprised of an anchor molecule, a first binding partner is covalently bonded to the ligand at a ratio of 1-4 molecules of the first binding partner per one molecule of the ligand; a second binding partner is covalently bonded to the anchor molecule whereby the second binding partner is immobilized to the delivery vehicle; and the first binding partner is covalently bonded to the second binding partner.
A61K 47/68 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p.ex. les supports ou les additifs inertes; Agents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p.ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un anticorps, une immunoglobuline ou son fragment, p.ex. un fragment Fc
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p.ex. les supports ou les additifs inertes; Agents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p.ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p.ex. émulsion, particule, complexe d’inclusion, stent ou kit
56.
MARIBAVIR ISOMERS, COMPOSITIONS, METHODS OF MAKING AND METHODS OF USING
The invention relates to novel compositions and methods of using maribavir which enhance its effectiveness in medical therapy, as well as to maribavir isomers and methods of use thereof for counteracting the potentially adverse effects of maribavir isomerization in vivo in the event it occurs.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p.ex. composés antiphlogistiques et pour le cœur
G01N 33/94 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir des narcotiques
G09B 19/00 - Enseignement non couvert par d'autres groupes principaux de la présente sous-classe
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
wherein each symbol is as described in the specification, or a salt thereof, is useful as an agent for the prophylaxis or treatment of narcolepsy.
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
C07D 413/04 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle contenant deux hétérocycles liés par une liaison directe de chaînon cyclique à chaînon cyclique
C07D 519/00 - Composés hétérocycliques contenant plusieurs systèmes de plusieurs hétérocycles déterminants condensés entre eux ou condensés avec un système carbocyclique commun non prévus dans les groupes ou
58.
TREATMENT OF GASTROINTESTINAL BLEEDING IN PATIENTS WITH SEVERE VON WILLEBRAND DISEASE BY ADMINISTRATION OF RECOMBINANT VWF
The present invention relates to a method for treating gastrointestinal bleeding in a subject with severe von Willebrand Disease comprising administering to the subject at least one dose of recombinant von Willebrand Factor (rVWF) ranging from about 40 IU/kg to about 100 IU/kg, wherein the first dose further comprises recombinant Factor VIII (rFVIII).
The present invention provides a compound having a lysine-specific demethylase-1 inhibitory action, and useful as a medicament such as a prophylactic or therapeutic agent for schizophrenia, developmental disorders, particularly diseases having intellectual disability (e.g., autistic spectrum disorders, Rett syndrome, Down's syndrome, Kabuki syndrome, fragile X syndrome, Kleefstra syndrome, neurofibromatosis type 1, Noonan syndrome, tuberous sclerosis), neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease, spinocerebellar degeneration (e.g., dentatorubral pallidoluysian atrophy) and Huntington's disease), epilepsy (e.g., Dravet syndrome) or drug dependence, and the like. A compound represented by the formula
The present invention provides a compound having a lysine-specific demethylase-1 inhibitory action, and useful as a medicament such as a prophylactic or therapeutic agent for schizophrenia, developmental disorders, particularly diseases having intellectual disability (e.g., autistic spectrum disorders, Rett syndrome, Down's syndrome, Kabuki syndrome, fragile X syndrome, Kleefstra syndrome, neurofibromatosis type 1, Noonan syndrome, tuberous sclerosis), neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease, spinocerebellar degeneration (e.g., dentatorubral pallidoluysian atrophy) and Huntington's disease), epilepsy (e.g., Dravet syndrome) or drug dependence, and the like. A compound represented by the formula
The present invention provides a compound having a lysine-specific demethylase-1 inhibitory action, and useful as a medicament such as a prophylactic or therapeutic agent for schizophrenia, developmental disorders, particularly diseases having intellectual disability (e.g., autistic spectrum disorders, Rett syndrome, Down's syndrome, Kabuki syndrome, fragile X syndrome, Kleefstra syndrome, neurofibromatosis type 1, Noonan syndrome, tuberous sclerosis), neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease, spinocerebellar degeneration (e.g., dentatorubral pallidoluysian atrophy) and Huntington's disease), epilepsy (e.g., Dravet syndrome) or drug dependence, and the like. A compound represented by the formula
wherein each symbol is as defined in the present specification, or a salt thereof.
C07D 417/14 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre et d'azote comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant au moins trois hétérocycles
C07D 231/12 - Composés hétérocycliques contenant des cycles diazole-1, 2 ou diazole-1, 2 hydrogéné non condensés avec d'autres cycles comportant deux ou trois liaisons doubles entre chaînons cycliques ou entre chaînons cycliques et chaînons non cycliques avec uniquement des atomes d'hydrogène, des radicaux hydrocarbonés ou des radicaux hydrocarbonés substitués, liés directement aux atomes de carbone du cycle
C07D 231/14 - Composés hétérocycliques contenant des cycles diazole-1, 2 ou diazole-1, 2 hydrogéné non condensés avec d'autres cycles comportant deux ou trois liaisons doubles entre chaînons cycliques ou entre chaînons cycliques et chaînons non cycliques avec des hétéro-atomes ou avec des atomes de carbone comportant trois liaisons à des hétéro-atomes, avec au plus une liaison à un halogène, p.ex. radicaux ester ou nitrile, liés directement aux atomes de carbone du cycle
C07D 277/56 - Atomes de carbone comportant trois liaisons à des hétéro-atomes avec au plus une liaison à un halogène
C07D 307/79 - Benzo [b] furannes; Benzo [b] furannes hydrogénés avec uniquement des atomes d'hydrogène, des radicaux hydrocarbonés ou des radicaux hydrocarbonés substitués, liés directement aux atomes de carbone de l'hétérocycle
C07D 307/81 - Radicaux substitués par des atomes d'azote ne faisant pas partie d'un radical nitro
C07D 333/38 - Atomes de carbone comportant trois liaisons à des hétéro-atomes avec au plus une liaison à un halogène, p.ex. radicaux ester ou nitrile
C07D 407/12 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'oxygène comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant deux hétérocycles liés par une chaîne contenant des hétéro-atomes comme chaînons
C07D 409/12 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre comme uniques hétéro-atomes du cycle contenant deux hétérocycles liés par une chaîne contenant des hétéro-atomes comme chaînons
C07D 409/14 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre comme uniques hétéro-atomes du cycle contenant au moins trois hétérocycles
C07D 413/12 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle contenant deux hétérocycles liés par une chaîne contenant des hétéro-atomes comme chaînons
C07D 413/14 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle contenant au moins trois hétérocycles
C07D 417/12 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre et d'azote comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant deux hétérocycles liés par une chaîne contenant des hétéro-atomes comme chaînons
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity.
A compound represented by the formula (I):
wherein each symbol is as described in the specification, or a salt thereof has an orexin type 2 receptor agonist activity, and is useful as an agent for the prophylaxis or treatment of narcolepsy.
A61K 31/407 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p.ex. guanéthidine ou rifamycines ayant des cycles à cinq chaînons avec un azote comme seul hétéro-atome d'un cycle, p.ex. sulpiride, succinimide, tolmétine, buflomédil condensés avec des systèmes hétérocycliques, p.ex. kétorolac, physostigmine
A61K 31/437 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p.ex. guanéthidine ou rifamycines ayant des cycles à six chaînons avec un azote comme seul hétéro-atome d'un cycle condensés en ortho ou en péri avec des systèmes hétérocycliques le système hétérocyclique contenant un cycle à cinq chaînons ayant l'azote comme hétéro-atome du cycle, p.ex. indolizine, bêta-carboline
The present disclosure relates to the treatment of cancer using a combination therapy comprising Compound 1 and/or tautomers thereof or a pharmaceutically acceptable salt or hydrate thereof, and one or more PARP inhibitors.
The present disclosure relates to the treatment of cancer using a combination therapy comprising Compound 1 and/or tautomers thereof or a pharmaceutically acceptable salt or hydrate thereof, and one or more PARP inhibitors.
A61K 31/519 - Pyrimidines; Pyrimidines hydrogénées, p.ex. triméthoprime condensées en ortho ou en péri avec des hétérocycles
A61K 31/454 - Pipéridines non condensées, p.ex. pipérocaïne contenant d'autres systèmes hétérocycliques contenant un cycle à cinq chaînons avec l'azote comme hétéro-atome du cycle, p.ex. pimozide, dompéridone
A61K 31/502 - Pyridazines; Pyridazines hydrogénées condensées en ortho ou en péri avec des systèmes carbocycliques, p.ex. cinnoline, phtalazine
The present disclosure provides, among other things, a cryopreservation medium comprising a cryoprotectant, an albumin, a disaccharide and a non-pyrogenic and isotonic crystalloid solution. The disclosure also provides, among other things, a cryopreservation medium for cryopreserving immune cells, the medium comprising: human serum albumin (HSA), sodium chloride, sodium gluconate, sodium acetate trihydrate, potassium chloride, magnesium chloride, dimethyl sulfoxide (DMSO), and a trehalose. The present disclosure also provides, a method of cryopreserving immune cells, transporting and subsequently administering such immune cells to a patient in need thereof.
The invention provides assay methods of detecting plasma protease C1 inhibitor (C1-INH) that binds plasma kallikrein, Factor XII, or both, and uses thereof for identifying subjects at risk for or suffering from a pKal-mediated or bradykinin-mediated disorder. Provided methods permit analysis of patients with plasma kallikrein-mediated angioedema (KMA), or other diseases mediated by pKal useful in the evaluation and treatment.
G01N 33/573 - Tests immunologiques; Tests faisant intervenir la formation de liaisons biospécifiques; Matériaux à cet effet pour enzymes ou isoenzymes
C07K 14/435 - Peptides ayant plus de 20 amino-acides; Gastrines; Somatostatines; Mélanotropines; Leurs dérivés provenant d'humains
C07K 16/38 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre des inhibiteurs de protéase de structure peptidique
C07K 16/40 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre des enzymes
C12Q 1/37 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismes; Compositions à cet effet; Procédés pour préparer ces compositions faisant intervenir une hydrolase faisant intervenir une peptidase ou une protéinase
G01N 33/68 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir des protéines, peptides ou amino-acides
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity. A compound represented by the formula (I): wherein each symbol is as described in the specification, or a salt thereof has an orexin type 2 receptor agonist activity, and is useful as an agent for the prophylaxis or treatment of narcolepsy.
A61P 3/04 - Anorexigènes; Médicaments de l'obésité
A61P 3/08 - Médicaments pour le traitement des troubles du métabolisme de l'homéostase du glucose
A61P 9/04 - Agents inotropes, c. à d. stimulants de la contraction cardiaque; Médicaments pour le traitement de l'insuffisance cardiaque
A61P 19/08 - Médicaments pour le traitement des troubles du squelette des maladies osseuses, p.ex. rachitisme, maladie de Paget
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p.ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
A61K 31/439 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p.ex. guanéthidine ou rifamycines ayant des cycles à six chaînons avec un azote comme seul hétéro-atome d'un cycle le cycle formant une partie d'un système cyclique ponté, p.ex. quinuclidine
The present disclosure provides muscle trophic engineered AAV capsid polypeptides derived from capsid polypeptides from AAV1, AAV6, AAV8 and AAV9 serotypes; nucleic acids encoding same, engineered capsid helper plasmids, engineered AAV vectors comprising the engineered AAV capsid polypeptides, transfected cells, transduced cells, and methods of making and using same.
Characterization of drug-drug interaction properties and pharmacological properties of maribavir is useful to inform potential drug-drug interactions and dosing strategies when administering with co-medications.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61K 31/4166 - 1,3-Diazoles ayant des groupes oxo liés directement à l'hétérocycle, p.ex. phénytoïne
A61K 31/513 - Pyrimidines; Pyrimidines hydrogénées, p.ex. triméthoprime ayant des groupes oxo liés directement à l'hétérocycle, p.ex. cytosine
A61K 31/55 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p.ex. guanéthidine ou rifamycines ayant des cycles à sept chaînons, p.ex. azélastine, pentylènetétrazole
67.
LARGE SCALE PRODUCTION OF RECOMBINANT ARYLSULFATASE A AND COMPOSITIONS THEREOF
The present invention provides, among other things, improved large-scale methods and compositions comprising recombinant arylsulfatase A (ASA) protein for enzyme replacement therapy, for example in treating Metachromatic Leukodystrophy Disease (MLD). In one aspect, the method of the present invention is for large-scale production of highly pure recombinant ASA with high cellular uptake, lysosomal targeting and bioactivity.
The present disclosure discloses compositions comprising maribavir, methods of providing the same, and compositions providing intermediates useful in providing maribavir.
A61K 31/7056 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p.ex. nucléosides, nucléotides contenant des cycles à cinq chaînons avec l'azote comme hétéro-atome d'un cycle
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 31/4184 - 1,3-Diazoles condensés avec des carbocycles, p.ex. benzimidazoles
The invention provides a method for treating or preventing an a-synucleinopathy in a subject in need thereof, the method comprising administering to the subject a fixed dose of 50-5,000 mg of an anti-α-synuclein antibody, or antigen-binding fragment thereof. Also provided are corresponding compositions and kits.
Provided herein, among other things, is a method of determining a transgene copy number in a biological sample, the method comprising: (a) extracting genomic DNA (gDNA) from the biological sample using an automated process, thus isolating gDNA; (b) performing digital droplet PCR (ddPCR) on the isolated gDNA using one or more primers to amplify a transgene of interest and to amplify a reference gene, thus normalizing gDNA input; and (c) determining transgene copy number in the biological sample.
C12Q 1/6881 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p.ex. amorces ou sondes pour le typage de tissu ou de cellule, p.ex. sondes d’antigène leucocytaire humain [HLA]
C12Q 1/6806 - Préparation d’acides nucléiques pour analyse, p.ex. pour test de réaction en chaîne par polymérase [PCR]
Provided herein are methods and kits for analyzing a biological sample obtained from a subject having, suspected of having, or being at risk for a disease associated with the contact activation system.
C12Q 1/6883 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p.ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique
C12Q 1/686 - Réaction en chaine par polymérase [PCR]
C12Q 1/6876 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p.ex. amorces ou sondes
G01N 33/50 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique
72.
GENE THERAPY OF HEMOPHILIA A USING VIRAL VECTORS ENCODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION
The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VIII polypeptide.
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 31/573 - Composés contenant des systèmes cycliques du cyclopenta[a]hydrophénanthrène; Leurs dérivés, p.ex. stéroïdes substitués en position 17 bêta par une chaîne à deux atomes de carbone, p.ex. prégnane ou progestérone substitués en position 21, p.ex. cortisone, dexaméthasone, prednisone ou aldostérone
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
G01N 33/68 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir des protéines, peptides ou amino-acides
G01N 33/86 - Analyse chimique de matériau biologique, p.ex. de sang ou d'urine; Test par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligands; Test immunologique faisant intervenir le temps de coagulation du sang
Disclosed are compounds of Formula (1), and pharmaceutically acceptable salts thereof, wherein α, β, m, R5, R6, R7, R9, R10, R11, Ra, Rb, X1, X2, X3, X4and X8 are defined in the specification. This disclosure also relates to materials and methods for preparing compounds of Formula (1), to pharmaceutical compositions which contain them, and to their use for treating diseases, disorders, and conditions associated with NLRP3.
C07D 491/052 - Systèmes condensés en ortho avec un seul atome d'oxygène comme hétéro-atome du cycle contenant de l'oxygène le cycle contenant de l'oxygène étant à six chaînons
C07D 491/107 - Systèmes condensés en spiro avec un seul atome d'oxygène comme hétéro-atome du cycle contenant de l'oxygène
Presented herein are systems and methods for automated data management for the development and/or production of a pharmaceutical product. In certain embodiments, the automated data management systems and methods provide for automated chemistry, manufacturing, and controls (CMC) management.
The invention provides, inter alia, methods of reducing gastrointestinal immune-related adverse events, such as colitis and diarrhea, in subjects undergoing an immune treatment, such as an immune oncology treatment, such as anti-CTLA4 antibody and anti-PD-1 antibody combination treatment for melanoma. In certain aspects, the methods encompass administering a therapeutically effective amount of a polypeptide that inhibits MAdCAM-integrin binding, such as an anti-α4β7 integrin antibody, such vedolizumab or a related antibody.
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
Disclosed herein are antibodies that bind to coronavirus spike protein (e.g., SARS-CoV-2 spike protein), and methods of use thereof (e.g., treatment of subjects infected with SARSCoV-2).
The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.
C07D 519/00 - Composés hétérocycliques contenant plusieurs systèmes de plusieurs hétérocycles déterminants condensés entre eux ou condensés avec un système carbocyclique commun non prévus dans les groupes ou
An object of the present invention is to provide an ever-better method for producing sinusoidal endothelial cells. The present invention provides a method for producing sinusoidal endothelial cells from sinusoidal endothelial progenitor cells, the method comprising the step of culturing the sinusoidal endothelial progenitor cells in a medium containing one or more substances selected from the interleukin 6 (IL-6) family, for example, oncostatin M (OSM), interleukin 6 (IL-6), or interleukin 11 (IL-11).
Methods for treating patients with autoimmune diseases such as myasthenia gravis (MG) by administering isolated anti-CD38 antibodies are disclosed. Also disclosed are unit dosage forms for the anti-CD38 antibodies used in treating patients with autoimmune diseases such as myasthenia gravis (MG).
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
A61K 38/00 - Préparations médicinales contenant des peptides
Methods of using polypeptides to modulate transforming growth factor-β (TGFβ) signaling (e.g., TGFβ receptors, antibodies or antigen-binding fragments thereof that specifically bind TGFβ or a TGFβ receptor) are provided. Compositions comprising the antibodies or fragments thereof and methods of using the same for treatment of diseases involving TGFβ activity are provided. Nucleic acids, recombinant expression vectors, host cells, antigen binding fragments, and pharmaceutical compositions comprising these antigen binding agents and fragments thereof are also disclosed. The invention also provides therapeutic methods for utilizing the TGFβ signaling modulators are provided herein.
C12N 5/0783 - Cellules T; Cellules NK; Progéniteurs de cellules T ou NK
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteurs; Vecteurs; Utilisation d'hôtes pour ceux-ci; Régulation de l'expression
82.
BUSINESS SUPPORT SYSTEM, BUSINESS SUPPORT METHOD, DATA MANAGEMENT PROGRAM, AND SEARCH PROGRAM
Provided is a mechanism for supporting work to respond to inquiries from relevant authorities. This business support system includes: a dividing unit for generating a plurality of sets of divided document data by dividing a plurality of inquiry items included in document data in an inquiry from a relevant authority, by inquiry item; a first acquiring unit for acquiring a plurality of sets of inputted divided document data in which response content with respect to inquiry content of each of the plurality of inquiry items has been input; a second acquiring unit for acquiring inputted document data, which are document data in one file format, generated from the plurality of acquired sets of inputted divided document data; and a storage unit for storing each of the plurality of acquired sets of inputted divided document data in a database in association with the inputted document data.
G06F 16/20 - Recherche d’informations; Structures de bases de données à cet effet; Structures de systèmes de fichiers à cet effet de données structurées, p.ex. de données relationnelles
G06F 16/383 - Recherche caractérisée par l’utilisation de métadonnées, p.ex. de métadonnées ne provenant pas du contenu ou de métadonnées générées manuellement utilisant des métadonnées provenant automatiquement du contenu
G06Q 50/26 - Services gouvernementaux ou services publics
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity. Provided is a compound of formula (I):
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity. Provided is a compound of formula (I):
The present invention provides a heterocyclic compound having an orexin type 2 receptor agonist activity. Provided is a compound of formula (I):
wherein each symbol is as described in the specification, or a salt thereof has an orexin type 2 receptor agonist activity, and is useful as an agent for the prophylaxis or treatment of narcolepsy.
C07D 413/04 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle contenant deux hétérocycles liés par une liaison directe de chaînon cyclique à chaînon cyclique
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
C07D 413/14 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle contenant au moins trois hétérocycles
The present disclosure provides recombinant antigen presenting cells and methods of use thereof in the culture and expansion of immune cells ex vivo. In some aspects, immune cells expanded through co-culture of the recombinant antigen presenting cells disclosed herein are administered to a subject to treat a disease or condition in the subject, e.g., to treat a cancer.
A syringe stabilizing apparatus has a base and a syringe support. The syringe support is vertically disposed above the base, elevating a fluid-filled portion of an infusion set vertically above the base and orienting a delivery end of the fluid-filled portion upwardly relative to a horizontal plane to take advantage of a gravitational effect on a fluid during delivery of the fluid from the fluid-filled portion to a patient. The syringe support comprises a first retainer and a selectively actuated tube clamp. The first retainer has an opening in which a rigid portion of the infusion set is received and retained therein without further user intervention. The selectively actuated tube clamp is operatively aligned with the first retainer wherein a flexible tube extending from the rigid portion of the infusion set extends through the tube clamp.
A61M 5/00 - Dispositifs pour faire pénétrer des agents dans le corps par introduction sous-cutanée, intravasculaire ou intramusculaire; Accessoires à cet effet, p.ex. dispositifs de remplissage ou de nettoyage, appuis-bras
A61M 5/315 - Pistons; Tiges de piston; Guidage, blocage, ou limitation des mouvements de la tige; Accessoires disposés sur la tige pour faciliter le dosage
A61M 25/02 - Dispositifs de maintien en position, p.ex. sur le corps
86.
COMPOSITIONS AND METHODS FOR TREATING ALPHA-SYNUCLEINOPATHIES
The invention provides a method for treating or preventing an α-synucleinopathy in a subject in need thereof, the method comprising administering to the subject a fixed dose of 50-5,000 mg of an anti-α-synuclein antibody, or antigen-binding fragment thereof. Also provided are corresponding compositions and kits. As an example, the antibody may be aslo452 ngl-3.
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p.ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
The present disclosure provides improved methods of producing engineered immune cells (e.g., CAR-T cells). The resulting engineered immune cells and compositions comprising the same are useful in treating various diseases, e.g., infection, autoimmune diseases, and tumors.
09 - Appareils et instruments scientifiques et électriques
35 - Publicité; Affaires commerciales
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Downloadable virtual goods, namely, software relating to
pharmaceutical preparations, medical devices, health care
and medical services for use in online virtual environments,
augmented reality, extended reality, and mixed reality
environments; downloadable digital files authenticated by
non-fungible tokens [NFTs]; downloadable software for
providing access to online virtual environments, augmented
reality, extended reality, and mixed reality environments
related to the following fields: pharmaceutical products,
medical devices, health care and medical services;
downloadable software for providing access to online virtual
environments, augmented reality, extended reality, and mixed
reality environments related to educational services in the
following fields: pharmaceutical products, medicines, health
care and medical services; downloadable software for
providing access to online virtual environments, augmented
reality, extended reality, and mixed reality environment
games; downloadable software for providing access to video
games. Providing online retail store services featuring virtual or
mixed reality software that may display virtual goods and
enables customers to purchase and store virtual goods
related to pharmaceutical preparations, medical devices,
health care and medical services for use in online virtual
environments, augmented reality, extended reality, and mixed
reality environments; Providing online marketplace services
featuring virtual or mixed reality software that may display
virtual goods and enables customers to purchase and store
virtual goods for buyers and sellers of virtual goods
related to pharmaceutical preparations, medical devices,
health care and medical services for use in online virtual
environments, augmented reality, extended reality, and mixed
reality environments. Entertainment services, namely, providing online,
non-downloadable virtual goods related to the following
fields: pharmaceutical products, medical devices, health
care and medical services for use in for use in online
virtual environments, augmented reality, extended reality,
and mixed reality environments created for entertainment
purposes; Entertainment services, namely, providing online,
non-downloadable virtual environments, augmented reality,
extended reality, and mixed reality environment games;
entertainment services, namely, providing online,
non-downloadable video games; entertainment services,
namely, providing online virtual environments, augmented
reality, extended reality, and mixed reality environments in
which users can interact for recreational and business
purposes. Providing temporary use of non-downloadable software for
virtual goods related to pharmaceutical preparations,
medical devices, health care and medical services for use in
online virtual environments, augmented reality, extended
reality, and mixed reality environments; Providing temporary
use of non-downloadable software for providing access to
online virtual environments, augmented reality, extended
reality, and mixed reality environments related to the
following fields: pharmaceutical products, medical devices,
health care and medical services; providing temporary use of
non-downloadable software for providing access to online
virtual environments, augmented reality, extended reality,
and mixed reality environments related to educational
services in the following fields: medicines, pharmaceutical
products, health care and medical services.
89.
Sting modulator compounds, and methods of making and using
The present disclosure provides STING modulators/agonists, and methods of synthesis and methods for using for the prophylaxis or treatment of cancer and other STING-related diseases.
The present disclosure relates to a compound represented by the Formula (I):
wherein each symbol is as defined in the description, or a pharmaceutically acceptable salt thereof.
C07H 19/207 - Radicaux purine avec le radical saccharide estérifié par des acides phosphoriques ou polyphosphoriques les acides phosphoriques ou polyphosphoriques étant estérifiés par un autre composé hydroxylique, p.ex. les dinucléotides de la flavine-adénine ou de la nicotinamide-adénine
A container unit may be used to facilitate administrations of multiple medicinal fluids to a patient. A container unit may include a first container, a second container, and a carrier which holds the first container and the second container stationary relative to each other. The carrier may include a lip configured to engage a pooling device to secure the container unit to the pooling device. The carrier may also include a slot configured to engage an insert on the pooling device to guide the container unit as the container unit is secured to the pooling device. The carrier may also include a first portion and second portion with different shapes that are complementary to a shape of a port on the pooling device. The carrier may also include an extension which extends in a direction away from one of the first container to a level at least even with a stopper disposed in the first container. The container unit may include a lid including at least one rotation inhibitor configured to inhibit rotation of the lid about at least one axis. A plurality of container units may include container units having different volume containers while maintaining a congruent interface portions.
A61J 1/14 - Récipients spécialement adaptés à des fins médicales ou pharmaceutiques - Détails; Accessoires à cet effet
B65D 71/50 - Paquets d'objets maintenus ensemble par des éléments d'emballage pour la commodité du stockage ou du transport, p.ex. paquets compartimentés pour le transport à la main de plusieurs réceptacles tels que des boîtes de bière ou des bouteilles de boisso; Balles de matériaux comprenant plusieurs objets maintenus ensemble uniquement de façon partielle par des éléments d'emballage formés autrement que par liage d'un flan
The present invention provides a method for measuring the amount of dystrophin in a sample, the method comprising the following steps. (1) A step for bringing dystrophin in a sample into contact with a dystrophin-capture antibody and thereby forming a composite I comprising the dystrophin and the capture antibody. (2) A step for bringing the composite I into contact with a dystrophin-detection antibody and thereby forming a composite II comprising the dystrophin, the capture antibody, and the detection antibody. (3) A step for dissociating and collecting a composite III comprising (i) the detection antibody or (ii) the dystrophin and the detection antibody from the composite II. (4) A step for measuring the amount of (i) the detection antibody or (ii) the composite III and thereby calculating the amount of the dystrophin in the sample.
G01N 33/53 - Tests immunologiques; Tests faisant intervenir la formation de liaisons biospécifiques; Matériaux à cet effet
G01N 33/543 - Tests immunologiques; Tests faisant intervenir la formation de liaisons biospécifiques; Matériaux à cet effet avec un support insoluble pour l'immobilisation de composés immunochimiques
Provided herein are compounds that inhibit pKal, a serine protease whose activity is responsible for proteolytically cleaving kininogen and generating the potent vasodilator and pro-inflammatory peptide bradykinin, which can lead to painful and debilitating inflammatory attacks (e.g., edema). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating pKal-related diseases and disorders (e.g., edema) with the compounds in a subject, by administering the compounds and/or compositions described herein.
C07D 417/04 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre et d'azote comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant deux hétérocycles liés par une liaison directe de chaînon cyclique à chaînon cyclique
C07D 417/14 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre et d'azote comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant au moins trois hétérocycles
The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.
C07D 519/00 - Composés hétérocycliques contenant plusieurs systèmes de plusieurs hétérocycles déterminants condensés entre eux ou condensés avec un système carbocyclique commun non prévus dans les groupes ou
94.
THERAPEUTIC METHODS USING CONSTRAINED CONDITIONALLY ACTIVATED BINDING PROTEINS
The invention relates to COnditional Bispecific Redirected Activation constructs, or COBRAs, that are administered in an active pro-drug format. Upon exposure to tumor proteases, the constructs are cleaved and activated, such that they can bind both tumor target antigens (TTAs) as well as CD3, thus recruiting T cells expressing CD3 to the tumor, resulting in treatment.
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
The invention relates to COnditional Bispecific Redirected Activation constructs, or COBRAs, that are administered in an active pro-drug format. Upon exposure to tumor proteases, the constructs are cleaved and activated, such that they can bind both tumor target antigens (TTAs) as well as CD3, thus recruiting T cells expressing CD3 to the tumor, resulting in treatment. In some embodiments, the tumor target antigen is B7H3.
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
09 - Appareils et instruments scientifiques et électriques
35 - Publicité; Affaires commerciales
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Downloadable virtual goods, namely, software relating to
pharmaceutical preparations, medical devices, health care
and medical services for use in online virtual environments,
augmented reality, extended reality, and mixed reality
environments; downloadable digital files authenticated by
non-fungible tokens [NFTs]; downloadable software for
providing access to online virtual environments, augmented
reality, extended reality, and mixed reality environments
related to pharmaceutical preparations, medical devices,
health care and medical services; downloadable software for
providing access to online virtual environments, augmented
reality, extended reality, and mixed reality environments
related to educational services in the field of
pharmaceuticals, medicines, health care and medical
services; downloadable software for providing access to
online virtual environments, augmented reality, mixed
reality, and extended reality environment games;
downloadable software for providing access to video games. Providing online retail store services featuring virtual or
mixed reality software that may display virtual goods and
enables customers to purchase and store virtual goods
related to pharmaceutical preparations, medical devices,
health care and medical services for use in online virtual
environments, augmented reality, extended reality, and mixed
reality environments; Providing online marketplace services
featuring virtual or mixed reality software that may display
virtual goods and enables customers to purchase and store
virtual goods for buyers and sellers of virtual goods
related to pharmaceutical preparations, medical devices,
health care and medical services for use in online virtual
environments, augmented reality, extended reality, and mixed
reality environments. Entertainment services, namely, providing online,
non-downloadable virtual goods related to pharmaceutical
preparations, medical devices, health care and medical
services for use in for use in online virtual environments,
augmented reality, extended reality, and mixed reality
environments created for entertainment purposes;
Entertainment services, namely, providing online,
non-downloadable virtual environments, augmented reality,
extended reality, and mixed reality environment games;
entertainment services, namely, providing online,
non-downloadable video games; entertainment services,
namely, providing online virtual environments, augmented
reality, extended reality, and mixed reality environments in
which users can interact for recreational and business
purposes. Providing temporary use of non-downloadable software for
virtual goods related to pharmaceutical preparations,
medical devices, health care and medical services for use in
online virtual environments, augmented reality, extended
reality, and mixed reality environments; Providing temporary
use of non-downloadable software for providing access to
online virtual environments, augmented reality, extended
reality, and mixed reality environments related to
pharmaceutical preparations, medical devices, health care
and medical services; Providing temporary use of non-
downloadable software for providing access to online virtual
environments, augmented reality, extended reality, and mixed
reality environments related to educational services in the
field of medicines, pharmaceuticals, health care and medical
services.
97.
VIRAL INHIBITORS, THE SYNTHESIS THEREOF, AND INTERMEDIATES THERETO
The present disclosure discloses compositions comprising maribavir, methods of providing the same, and compositions providing intermediates useful in providing maribavir.
The present disclosure provides oligonucleotides and modified oligonucleotides, lipid nanoparticles comprising oligonucleotides and modified oligonucleotides, methods for reducing or suppressing an immune response or for increasing an expression of protein encoded by a nucleic acid included in the lipid nanoparticles in a subject receiving nucleic acid therapy, and methods for treating or preventing a genetic disorder in a subject.
C12N 15/117 - Acides nucléiques présentant des propriétés immunomodulatrices, p.ex. contenant des motifs CpG
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c. à d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c. à d. autres que des liaisons 3'-5' phosphodiester
C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p.ex. co-transformation utilisant la micro-encapsulation, p.ex. utilisant des vésicules liposomiques
Provided is a method for activating T cells comprising the step of activating T cells in a medium containing a CD3 agonist and/or a CD28 agonist for more than 36 hours and less than 48 hours; a method for producing genetically modified T cells, comprising the steps of activating T cells according to the activation method, introducing an exogenous gene into the activated T cells, and culturing the T cells into which the exogenous gene has been introduced; a cell population comprising genetically modified T cells obtained by the production method; and a matrix of mobile polymer chains or a bead supporting a CD3 agonist and/or a CD28 agonist, wherein the matrix or bead is used for being added to a medium and subjecting T cells to an activation step for more than 36 hours and less than 48 hours.
C12N 5/0783 - Cellules T; Cellules NK; Progéniteurs de cellules T ou NK
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
The invention provides methods for the treatment of chronic pouchitis comprising administering an anti-α4β7 antibody, e.g., vedolizumab, to a human subject in need thereof.
C07K 16/28 - Immunoglobulines, p.ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
A61K 31/496 - Pipérazines non condensées contenant d'autres hétérocycles, p.ex. rifampine, thiothixène
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps