Non-human animal cells and non-human animals comprising a humanized ACE2 locus and a humanized TMPRSS locus, and methods of using such non-human animal cells and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized ACE2 locus and a humanized TMPRSS locus express a human ACE2 protein or a chimeric ACE2 protein, fragments of which are from human ACE2; and a human TMPRSS or chimeric TMPRSS protein, fragments of which are from human TMPRSS. Methods are also provided for using such non-human animals comprising a humanized ACE2 locus and a humanized TMPRSS locus to assess in vivo ACE2 activity, e.g., coronavirus infection and/or the treatment or prevention thereof.
B-cell maturation antigen (BCMA) is expressed on malignant plasma cells. The present invention provides methods for treating multiple myeloma using bispecific antibodies (bsAbs) that bind to both BCMA and CD3 and activate T cells via the CD3 complex in the presence of BCMA-expressing tumor cells. In certain embodiments, the bispecific antigen-binding molecules of the present invention are capable of inhibiting the growth of tumors expressing BCMA.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
3.
ANTI-TFR:ACID SPHINGOMYELINASE FOR TREATMENT OF ACID SPHINGOMYELINASE DEFICIENCY
Multidomain therapeutic proteins comprising a TfR-binding delivery domain fused to an acid sphingomyelinase polypeptide and nucleic acid constructs and compositions that allow insertion of a multidomain therapeutic protein coding sequence into a target genomic locus such as an endogenous ALB locus and/or expression of the multidomain therapeutic protein coding sequence are provided. The multidomain therapeutic proteins and nucleic acid constructs and compositions can be administered to cells, populations of cells, or subjects and can be used in methods of integration of a multidomain therapeutic protein nucleic acid into a target genomic locus, methods of expression of a multidomain therapeutic protein in a cell, methods of treating acid sphingomyelinase deficiency in a subject, and methods of preventing or reducing the onset of a sign or symptom of acid sphingomyelinase deficiency in a subject.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C12N 9/16 - Hydrolases (3.) acting on ester bonds (3.1)
The present invention provides antibodies and antigen-binding fragments of antibodies that bind to leptin receptor (LEPR), and methods of using the same. According to certain embodiments, the invention includes antibodies and antigen-binding fragments of antibodies that bind LEPR and activate LEPR signaling. In other embodiments, the invention includes antibodies and antigen-binding fragments of antibodies that bind to LEPR and enhance sensitization of LEPR to an antigen. In certain embodiments, the invention includes antibodies and antigen-binding fragments of antibodies that bind LEPR in the presence and absence of leptin. In certain embodiments, the invention includes antibodies and antigen-binding fragments of antibodies that induce signaling in cells expressing LEPR mutants that otherwise exhibit defective or impaired signaling in the presence of leptin. The antibodies and antigen-binding fragments of the present invention are useful for the treatment of lipodystrophies and other diseases and disorders associated with or caused by leptin deficiency or leptin resistance.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
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.: Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy.
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
Provided herein are compounds, compositions, and methods for the treatment of diseases and disorders associated with influenza, including VX-787 and derivatives thereof, and protein (e.g., antibody) drug conjugates thereof.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
A61K 47/65 - Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
A61P 31/16 - Antivirals for RNA viruses for influenza or rhinoviruses
Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized MYOC locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized MYOC locus express a human myocilin protein or a chimeric myocilin protein, fragments of which are from human myocilin. Methods are provided for using such non-human animals comprising a humanized MYOC locus to assess in vivo efficacy of human-myocilin-targeting reagents and reagents for treating glaucoma.
A01K 67/0275 - Genetically modified vertebrates, e.g. transgenic
C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
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
The present disclosure provides bispecific antigen-binding molecules that bind to PD-L1 and CD28 (PD-L1×CD28). In certain embodiments, the present disclosure provides for a bispecific PD-L1×CD28 antigen-binding molecule (or antibody) or antigen-binding fragment thereof comprising a first antigen-binding domain that specifically binds CD28, and a second antigen-binding domain that specifically binds PD-L1. In certain embodiments, the bispecific antigen-binding molecules of the present disclosure bind CD28 on T-cells with the first antigen-binding domain and PD-L1 expressed on tumor cells or antigen presenting cells with the second antigen-binding domain. In certain embodiments, the bispecific antigen-binding molecules are capable of inhibiting growth of a tumor. The bispecific antigen-binding molecules of this disclosure are useful for treatment of cancer.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
Ophthalmic formulations of a vascular endothelial growth factor (VEGF)-specific fusion protein antagonist are provided suitable for intravitreal administration to the eye. The ophthalmic formulations include a stable liquid formulation and a lyophilizable formulation. Preferably, the protein antagonist has an amino acid sequence of SEQ ID NO:4.
C07K 14/71 - ReceptorsCell surface antigensCell surface determinants for growth factorsReceptorsCell surface antigensCell surface determinants for growth regulators
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 9/19 - Particulate form, e.g. powders lyophilised
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
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
28.
ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTION FOR TREATMENT OF POMPE DISEASE
ALBALB locus and/or expression of the multidomain therapeutic protein (e.g., GAA fusion protein) coding sequence are also provided. The nucleic acid constructs and compositions can be used in methods of integration of a multidomain therapeutic protein (e.g., GAA fusion protein) nucleic acid into a target genomic locus, methods of expression of a multidomain therapeutic protein (e.g., GAA fusion protein) in a cell, methods of reducing glycogen accumulation, methods of treating Pompe disease or GAA deficiency in a subject, and method of preventing or reducing the onset of a sign or symptom of Pompe disease in a subject, including neonatal cells and subjects.
29.
METHODS FOR REDUCING THE LIKELIHOOD OF AND TREATING GRAFT-VERSUS-HOST DISEASE
The present disclosure provides a method for reducing the likelihood of or treating graft-vs-host disease in a subject by performing assays on GI biopsies from the subject to measure cell numbers and density ratios and by administering to the subject a pharmaceutically effective amount of one or more immunosuppressant agents.
The present disclosure provides methods for treating, reducing the severity, or inhibiting the growth of prostate cancer or metastatic castration-resistant prostate cancer. The methods of the present disclosure comprise administering to a subject in need thereof a therapeutically effective amount of a bispecific antibody or antigen-binding fragment thereof that specifically binds prostate- specific membrane antigen (PSMA) and CD28 as monotherapy or in combination with an anti-PDl antibody.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
35.
Stabilized Formulations Containing Anti-BCMA x Anti-CD3 Bispecific Antibodies
The present invention provides stable liquid pharmaceutical formulations comprising a human bispecific antibody that specifically binds to human BCMA and human CD3. In certain embodiments, the formulations contain, in addition to the bispecific antibody, a buffer, a surfactant, and a sugar. The pharmaceutical formulations of the present invention exhibit a substantial degree of antibody stability upon stress and storage.
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharidesDerivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
36.
BISPECIFIC PD-L1x4-1BB ANTIBODIES AND METHODS OF USE THEREOF
The present disclosure provides bispecific antigen-binding molecules that bind to PD-L1 and 4-1BB (PD-L1×4-1BB). In certain embodiments, the present disclosure provides a bispecific PD-L1×4-1BB antibody or antigen-binding fragment thereof comprising a first antigen-binding domain that specifically binds 4-1BB, and a second antigen-binding domain that specifically binds PD-L1. In certain embodiments, the bispecific antibodies disclosed herein bind 4-1BB on T-cells with the first antigen-binding domain and PD-L1 expressed on tumor cells or antigen presenting cells with the second antigen-binding domain. In certain embodiments, the antibodies of the present disclosure are useful in treatment of a cancer.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
Nucleic acid constructs and compositions that allow insertion of a multidomain therapeutic protein (e.g., GAA fusion protein) coding sequence into a target genomic locus such as an endogenous ALB locus and/or expression of the multidomain therapeutic protein (e.g., GAA fusion protein) coding sequence are also provided. The nucleic acid constructs and compositions can be used in methods of integration of a multidomain therapeutic protein (e.g., GAA fusion protein) nucleic acid into a target genomic locus, methods of expression of a multidomain therapeutic protein (e.g., GAA fusion protein) in a cell, methods of reducing glycogen accumulation, methods of treating Pompe disease or GAA deficiency in a subject, and method of preventing or reducing the onset of a sign or symptom of Pompe disease in a subject, including neonatal cells and subjects.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 38/47 - Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
Provided herein are method of treating cancer using bispecific antigen-binding molecules that bind to Mucin 16 (MUC16) and CD3. According to certain embodiments, the antibodies useful herein bind human MUC16 with high affinity and bind CD3 to induce human T cell proliferation. According to certain embodiments, bispecific antigen-binding molecules comprising a first antigen-binding domain that specifically binds human CD3, and a second antigen-binding molecule that specifically binds human MUC16 are particularly useful herein. In certain embodiments, the bispecific antigen-binding molecules in combination with an anti-4-1BB agonist are capable of inhibiting the growth of tumors expressing MUC16, for example, ovarian tumors. The bispecific antigen-binding molecules in combination with an anti-4-1BB agonist are useful for the treatment of diseases and disorders in which an upregulated or induced targeted immune response is desired and/or therapeutically beneficial, for example, in the treatment of various cancers.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61B 6/00 - Apparatus or devices for radiation diagnosisApparatus or devices for radiation diagnosis combined with radiation therapy equipment
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61K 51/10 - Antibodies or immunoglobulinsFragments thereof
C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
39.
CANCER THERAPIES TARGETING VISTA AS MONOTHERAPY OR IN COMBINATION WITH ANTI-PD1 ANTIBODY TO TREAT CANCER
Provided herein are doses and dosing regimens for administration of an anti-VISTA antibody for treating cancer in human subjects. The anti-VISTA antibody may be administered as a monotherapy or in combination with an anti-PD-1 antibody.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
40.
USE OF BGH-SV40L TANDEM POLYA TO ENHANCE TRANSGENE EXPRESSION DURING UNIDIRECTIONAL GENE INSERTION
Unidirectional SV40 late polyadenylation signals and combinations of such unidirectional SV40 late polyadenylation signals with other polyadenylation signals such as bovine growth hormone (BGH) polyadenylation signals are provided. The polyadenylation signals can be used in nucleic acid constructs and compositions that allow insertion of a coding sequence for a polypeptide interest into a target genomic locus and/or expression of the polypeptide interest. The nucleic acid constructs and compositions can be used in methods of integration of a coding sequence for a polypeptide interest into a target genomic locus and methods of expression of a polypeptide interest in a cell.
41.
ANTI-TFR: ACID SPHINGOMYELINASE FOR TREATMENT OF ACID SPHINGOMYELINASE DEFICIENCY
Multidomain therapeutic proteins comprising a TfR-binding delivery domain fused to an acid sphingomyelinase polypeptide and nucleic acid constructs and compositions that allow insertion of a multidomain therapeutic protein coding sequence into a target genomic locus such as an endogenous ALB locus and/or expression of the multidomain therapeutic protein coding sequence are provided. The multidomain therapeutic proteins and nucleic acid constructs and compositions can be administered to cells, populations of cells, or subjects and can be used in methods of integration of a multidomain therapeutic protein nucleic acid into a target genomic locus, methods of expression of a multidomain therapeutic protein in a cell, methods of treating acid sphingomyelinase deficiency in a subject, and methods of preventing or reducing the onset of a sign or symptom of acid sphingomyelinase deficiency in a subject.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/00 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
C12N 15/02 - Preparation of hybrid cells by fusion of two or more cells, e.g. protoplast fusion
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
42.
METHODS OF TREATING CLEAR CELL RENAL CELL CARCINOMA WITH BISPECIFIC ANTI-PSMA X ANTI-CD28 ANTIBODIES
The present disclosure provides methods for treating, reducing the severity, or inhibiting the growth of clear cell renal cell carcinoma (ccRCC) or metastatic ccRCC. The methods of the present disclosure comprise administering to a subject in need thereof a therapeutically effective amount of a bispecific antibody or antigen-binding fragment thereof that specifically binds prostate-specific membrane antigen (PSMA) and CD28.
C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
05 - Pharmaceutical, veterinary and sanitary products
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Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
49.
Treatment Of Hyperglycemia With Solute Carrier Family 39 Member 5 (SLC39A5) Inhibitors
The present disclosure provides methods of treating subjects having increased serum glucose level and/or hyperglycemia, methods of identifying subjects having an increased risk of developing increased serum glucose level and/or hyperglycemia, and methods of detecting human Solute Carrier Family 39 Member 5 variant nucleic acid molecules and variant polypeptides.
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
C07K 14/435 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
C12Q 1/6876 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
C12Q 1/6897 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
G01N 33/66 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving blood sugars, e.g. galactose
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 33/74 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving hormones
50.
METHODS OF EFFECTING A HEMODYNAMIC CHANGE BY ADMINISTERING AN ANTI-NPR1 ANTIBODY
The present disclosure is related to methods of reducing blood pressure or effecting a hemodynamic change in a subject, the methods comprising administering to the subject a pharmaceutical composition comprising an agonist of natriuretic peptide receptor 1 (NPR1) (e.g., an agonist anti-NPR1 antibody).
C07K 1/22 - Affinity chromatography or related techniques based upon selective absorption processes
C07K 1/36 - ExtractionSeparationPurification by a combination of two or more processes of different types
C07K 16/22 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C12N 5/00 - Undifferentiated human, animal or plant cells, e.g. cell linesTissuesCultivation or maintenance thereofCulture media therefor
C12N 15/66 - General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligationUse of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
C12P 21/02 - Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
C12P 21/06 - Preparation of peptides or proteins produced by the hydrolysis of a peptide bond, e.g. hydrolysate products
The present invention generally pertains to methods of characterizing non-consensus glycosylation sites of a protein. In particular, the present invention pertains to the use of high-throughput automated processes through digestion, enrichment of glycopeptides by liquid chromatography-mass spectrometry for identifying identification of non-consensus glycosylation sites.
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
The present invention provides methods to identify and quantify empty, partial and full capsids. The capsids can be purified from a crude AAV sample using affinity chromatography and eluted from the solid support. The purified capsids can then be subjected to density gradient equilibrium analytical ultracentrifugation (DGE-AUC) for characterization and quantitation of capsid content.
G01N 15/04 - Investigating sedimentation of particle suspensions
B01D 15/38 - Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups , e.g. affinity, ligand exchange or chiral chromatography
C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof
Disclosed are methods of quantifying multiple quality attributes, such as post translational modifications, of multiple samples in a single mass spectrometry (MS) run, including contacting two or more samples with a digesting solution under conditions sufficient to digest samples, wherein each sample is digested separately and the digesting solution is a Tris-free buffer solution; contacting each of the two or more digested samples with a specific Tandem Mass Tag (TMT) labeling reagent under conditions sufficient to label peptides within each of the digested samples with the specific TMT labeling reagent; quenching labeling of peptides within each of the two or more digested samples; combining equal volumes of the two or more labeled, digested samples into a single combined sample solution; and analyzing the single combined sample solution by targeted mass spectral analysis, thereby allowing multiple quality attributes of the two or more samples to be quantified in a single mass spectrometry (MS) run.
UNBIASED AND HIGH-THROUGHPUT IDENTIFICATION AND QUANTIFICATION OF HOST CELL PROTEIN IMPURITIES BY AUTOMATED ITERATIVE LC-MS/MS (HCP-AIMS) FOR THERAPEUTIC PROTEIN DEVELOPMENT
The present disclosure generally pertains to methods of identifying and quantitating host cell proteins (HCPs) in therapeutic protein development. In particular, the present invention generally pertains to methods of liquid chromatography-tandem mass spectrometry (LC-MS/MS) for unbiased identification and sensitive quantitation of HCPs in therapeutic protein development.
Compositions and methods for inserting a nucleic acid encoding a polypeptide of interest into a target genomic locus in a neonatal cell, a population of neonatal cells, or a neonatal subject or for expressing a nucleic acid encoding a polypeptide of interest in a neonatal cell, a population of neonatal cells, or a neonatal subject are provided. Also provided are neonatal cells or populations of neonatal cells comprising a nucleic acid construct comprising a coding sequence for a polypeptide of interest inserted into a target genomic locus.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
C12N 15/90 - Stable introduction of foreign DNA into chromosome
58.
METHODOLOGY TO CHARACTERIZE EMPTY-FULL RATIO IN CRUDE AAV SAMPLES
The present invention provides methods to identify and quantify empty, partial and full capsids. The capsids can be purified from a crude AAV sample using affinity chromatography and eluted from the solid support. The purified capsids can then be subjected to density gradient equilibrium analytical ultracentrifugation (DGE-AUC) for characterization and quantitation of capsid content.
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment of auditory, hematological, gastrointestinal, neuromuscular, muscular, cardiovascular, metabolic, immunological, inflammatory, oncological, ophthalmological, neurological, musculoskeletal, bone, connective tissue, dermatological, infectious, autoimmune, nephrological, hepatological, allergic, genetic, respiratory, viral, and pulmonary diseases and disorders; pharmaceutical preparations for the treatment of pain; pharmaceutical preparations for the treatment of obesity; pharmaceutical preparations for immunotherapy treatment, cell therapy, and gene therapy
64.
BOTULINUM NEUROTOXIN-SPECIFIC CAPTURE AGENTS, COMPOSITIONS, AND METHODS OF USING AND MAKING
The present application provides stable peptide-based Botulinum neurotoxin (BoNT) serotype A capture agents and methods of use as detection and diagnosis agents and in the treatment of diseases and disorders. The application further provides methods of manufacturing BoNT serotype A capture agents using iterative on-bead in situ click chemistry.
The present invention relates to regimens for the treatment of angiogenic eye disorders such as nAMD, DR and DME characterized by high doses of aflibercept extended periods between doses.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 9/00 - Medicinal preparations characterised by special physical form
The present invention provides monoclonal antibodies that bind to the Factor XII (FXII) protein, and methods of use thereof. In various embodiments of the invention, the antibodies are fully human antibodies that bind to FXII and to the activated form of FXII (FXIIa). In some embodiments, the antibodies of the invention are useful for inhibiting or neutralizing FXII activity, thus providing a means of treating or preventing a disease, disorder or condition associated with thrombosis in humans.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
Methods and systems for detecting abnormal karyotypes are disclosed. An example method can comprise determining read coverage data, allele balance distributions of heterozygous SNPs, and chromosomal segments where heterozygosity is not observed. The methods and systems can then determine one or more metrics which can be indicative of abnormal karyotype(s).
C12Q 1/6881 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for tissue or cell typing, e.g. human leukocyte antigen [HLA] probes
G16B 20/00 - ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
G16B 40/00 - ICT specially adapted for biostatisticsICT specially adapted for bioinformatics-related machine learning or data mining, e.g. knowledge discovery or pattern finding
Systems and methods for motor assessment including receiving first sensed signals in response to a first motor assessment performed using a first test device, receiving second sensed signals in response to a second motor assessment different than the first motor assessment and performed using the first test device, performing noise reduction for the first sensed signals and the second sensed signals, performing position normalization for the first sensed signals and the second sensed signals, generating transformed signals based on the noise reduction and the position normalization for the first sensed signals and the second sensed signals, extracting features based on the transformed signals, providing the extracted features to a machine learning model trained to output a motor assessment based prediction based on the transformed signals; and/or receiving the motor assessment based prediction from the machine learning model.
Embodiments disclosed herein are directed to systems and methods for motor assessment and include receiving first sensed signals in response to a first motor assessment performed using a first test device, receiving second sensed signals in response to a second motor assessment different than the first motor assessment and performed using the first test device, performing noise reduction for the first sensed signals and the second sensed signals, performing position normalization for the first sensed signals and the second sensed signals, generating transformed signals based on the noise reduction and the position normalization for the first sensed signals and the second sensed signals, extracting features based on the transformed signals, providing the extracted features to a machine learning model trained to output a motor assessment based prediction based on the transformed signals; and receiving the motor assessment based prediction from the machine learning model.
Non-human animal cells and non-human animals comprising a humanized Cacng1 locus and methods of using such non-human animal cells and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized Cacng1 locus express a human CACNG1 protein or fragments thereof.
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
72.
STABILIZED FORMULATIONS CONTAINING ANTI-BCMA X ANTI-CD3 BISPECIFIC ANTIBODIES
The present invention provides stable liquid pharmaceutical formulations comprising a human bispecific antibody that specifically binds to human BCMA and human CD3. In certain embodiments, the formulations contain, in addition to the bispecific antibody, a buffer, a surfactant, and a sugar. The pharmaceutical formulations of the present invention exhibit a substantial degree of antibody stability upon stress and storage.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
The present disclosure provides bispecific antigen-binding molecules that bind to PD-L1 and CD28 (PD-L1xCD28). In certain embodiments, the present disclosure provides fora bispecific PD-L1xCD28 antigen-binding molecule (or antibody) or antigen-binding fragment thereof comprising a first antigen-binding domain that specifically binds CD28, and a second antigen binding domain that specifically binds PD-L1. In certain embodiments, the bispecific antigen binding molecules of the present disclosure bind CD28 on T-cells with the first antigen-binding domain and PD-L1 expressed on tumor cells or antigen presenting cells with the second antigenbinding domain. In certain embodiments, the bispecific antigen-binding molecules are capable of inhibiting growth of a tumor. The bispecific antigen-binding molecules of this disclosure are useful for treatment of cancer.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
74.
Methods Of Treating Skin Cancer With Carboxypeptidase Vitellogenic Like (CPVL) Inhibitors
The present disclosure provides methods of treating a subject having skin cancer or preventing a subject from developing skin cancer, and methods of identifying subjects having an increased risk of developing skin cancer.
A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
75.
ANTI-HUMAN CACNG1 ANTIBODY-DRUG CONJUGATES AND USES THEREOF
Provided herein are antigen-binding protein-drug conjugates and compositions thereof that are useful, for example, for target-specific delivery of therapeutic moieties, e.g., testosterone analogs and/or derivatives. Dihydrotestosterone analogs, antibody-drug conjugates, and compositions which comprise glutaminyl-modified antibodies and dihydrotestosterone analogs and are provided.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
76.
BISPECIFIC PD-L1X4-1BB ANTIBODIES AND METHODS OF USE THEREOF
The present disclosure provides bispecific antigen-binding molecules that bind to PD-L1 and 4-1 BB (PD-L1x4-1 BB). In certain embodiments, the present disclosure provides a bispecific PD-L1x4-1 BB antibody or antigen-binding fragment thereof comprising a first antigen binding domain that specifically binds 4-1 BB, and a second antigen-binding domain that specifically binds PD-L1. In certain embodiments, the bispecific antibodies disclosed herein bind 4-1 BB on T-cells with the first antigen-binding domain and PD-L1 expressed on tumor cells or antigen presenting cells with the second antigen-binding domain. In certain embodiments, the antibodies of the present disclosure are useful in treatment of a cancer.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
Methods and compositions for treating centralized pain are disclosed. Methods and compositions for reducing pain or improving physical function are disclosed. In certain aspects, the subject has centralized pain and osteoarthritis of the knee and/or hip and the anti-NGF antibody is fasinumab.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
81.
OFF-TARGET PREDICTION METHOD FOR ANTIGEN-RECOGNITION MOLECULES BINDING TO MHC-PEPTIDE TARGETS
Computational systems and methods for predicting amino acid position(s) within a target peptide presented in a complex with a major histocompatibility complex (MHC) molecule (MHC-target peptide complex), the amino acid position(s) being involved in interacting with an antigen-recognition molecule that recognizes said MHC-target peptide complex, are presented herein. Computational systems and methods for estimating a number of off-target peptide(s) for an antigen-recognition molecule that recognizes a target peptide presented in a complex with a major histocompatibility complex (MHC) molecule (MHC-target peptide complex) is presented herein. Computational systems and methods for ranking potential target peptides to mitigate off-target toxicity are presented herein. Such computational systems and methods can streamline development of effective, well tolerated antigen-recognition molecules to treat diseases.
The present invention provides antibodies and antigen-binding fragments (e.g., human antibodies) that bind specifically to human Interleukin-36 receptor (IL36R). Methods for treating or preventing diseases mediated by IL36R (e.g., skin or colon inflammatory conditions such as palmo-plantar pustular psoriasis, palmoplantar pustulosis, generalized pustular psoriasis, ulcerative colitis or IBD) using the antibodies and fragments are also provided along with methods of making the antibodies and fragments.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
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 1/00 - Drugs for disorders of the alimentary tract or the digestive system
Provided herein are combinations comprising CRISPR/Cas systems, CtBP-interacting protein, and inhibitor of 53BP1 for use in enhancing homology-directed repair of CRISPR/Cas-mediated cleavage of a target DNA by an exogenous donor nucleic acid. Also provided are methods of using such combinations to make a targeted genetic modification in a cell by homology-directed repair of CRISPR/Cas-mediated cleavage at a target genomic locus in the cell.
The inventions provide methods for characterizing messenger RNAs (mRNA) and polyadenylation on mRNAs. The disclosed inventions are developed for characterizing mRNA and poly(A) tail length on mRNA samples. These inventions can be used in release testing of mRNA drug substrate (DS) or drug product (DP), and can provide direct read-out of mRNA integrity and poly(A) tail length. The inventions further provide methods of analyzing the condition of mRNAs in samples. Characterized mRNA samples obtained by all of the inventive methods also are part of the inventions disclosed herein.
The present disclosure provides anti-Platelet-Derived Growth Factor Subunit B (PDGF-B) antibodies, and antigen-binding fragments thereof, as well as methods of use of such antibodies, or antigen-binding fragments thereof, for treating a subject having pulmonary arterial hypertension (PAH).
A61P 43/00 - Drugs for specific purposes, not provided for in groups
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
Genetically modified non-human animals and methods and compositions for making and using them are provided, wherein the genetic modification comprises a deletion of the endogenous low affinity FcγR locus, and wherein the mouse is capable of expressing a functional FcRγ-chain. Genetically modified mice are described, including mice that express low affinity human FcγR genes from the endogenous FcγR locus, and wherein the mice comprise a functional FcRγ-chain. Genetically modified mice that express up to five low affinity human FcγR genes on accessory cells of the host immune system are provided.
The present invention provides antibodies that bind to interleukin-33 (IL-33) and methods of using the same. The invention includes antibodies that inhibit or attenuate IL-33-mediated signaling. The antibodies of the invention may function to block the interaction between IL-33 and ST2. Alternatively, certain antibodies of the invention inhibit or attenuate IL-33-mediated signaling without blocking the IL-33/ST2 interaction. According to certain embodiments of the invention, the antibodies are fully human antibodies that bind to human IL-33 with high affinity. The antibodies of the invention are useful for the treatment of diseases and disorders associated with IL-33 signaling and/or IL-33 cellular expression, such as inflammatory diseases, or allergic diseases.
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
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
88.
METHODS FOR PURIFYING FILLED ADENO-ASSOCIATED VIRUS CAPSIDS
The inventions provide methods of purifying full adeno-associated virus (AAV) capsids, wherein the method comprises the steps of (a) subjecting a sample comprising AAV capsids in buffer to anion exchange chromatography (AEX) to separate full AAV capsids from capsids that are not full (that is, partially-filled AAV capsids, nearly empty AAV capsids and empty AAV capsids) and form a first pool ("first AEX pass"), wherein the first pool is enriched in the ratio of full capsids to capsids that are not full; and (b) subjecting the first pool to anion exchange chromatography to form a second pool ("second AEX pass"), wherein the second pool is further enriched in the ratio of full capsids to capsids that are not full (that is, partially-filled AAV capsids, nearly empty AAV capsids and empty AAV capsids), wherein the enrichment in full capsids can be least 3 fold. Third, fourth and more AEX passes also can be undertaken. Depth filtration, single-pass tangential flow filtration and affinity exchange also can be included in the inventive methods, preferably undertaken prior to AEX. Enriched AAV preparations and drug products also are provided.
89.
CD20-PD1 BINDING MOLECULES AND METHODS OF USE THEREOF
The present disclosure relates to molecules capable of binding to both CD20 and PD1, as well as pharmaceutical compositions comprising such molecules and methods of use thereof.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
90.
METHODS FOR PURIFYING FILLED ADENO-ASSOCIATED VIRUS CAPSIDS
The inventions provide methods of purifying full adeno-associated virus (AAV) capsids, wherein the method comprises the steps of (a) subjecting a sample comprising AAV capsids in buffer to anion exchange chromatography (AEX) to separate full AAV capsids from capsids that are not full (that is, partially-filled AAV capsids, nearly empty AAV capsids and empty AAV capsids) and form a first pool (“first AEX pass”), wherein the first pool is enriched in the ratio of full capsids to capsids that are not full; and (b) subjecting the first pool to anion exchange chromatography to form a second pool (“second AEX pass”), wherein the second pool is further enriched in the ratio of full capsids to capsids that are not full (that is, partially-filled AAV capsids, nearly empty AAV capsids and empty AAV capsids), wherein the enrichment in full capsids can be least 3 fold. Third, fourth and more AEX passes also can be undertaken. Depth filtration, single-pass tangential flow filtration and affinity exchange also can be included in the inventive methods, preferably undertaken prior to AEX. Enriched AAV preparations and drug products also are provided.
In one instance, disclosed herein is an auto-injector, the auto-injector including: a housing; a container disposed within the housing, the container configured to store a medicament; a canister disposed within the housing, the canister configured to release a pressurized medium to expel the medicament from the container; a shroud coupled to the housing, the shroud configured to move between a first position and a second position relative to the housing to cause the pressurized medium to be released by the canister; and a mandrel assembly disposed within the housing, the mandrel assembly configured to urge the shroud outward relative to the housing toward the first position after the pressurized medium is released by the canister.
A non-human animal is genetically modified with sequence encoding a human or humanized MHC molecule or associated molecule, e.g., β2 microglobulin, and expression of the sequence by the non-human animal induces tolerance to the corresponding human HLA from which the human or human MHC molecule is derived. The tolerance exhibited by these non-human animals allows these animals to generate specific antibody responses to the corresponding human HLA when such HLA is presenting a peptide that is antigenic to the non-human animal. Such an antibody response, which specifically targets a pMHC complex of interest without binding to the MHC molecule may be useful in immunotherapeutic modalities that target a component of the immunological synapse which provides the specificity of that interaction.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A01K 67/0275 - Genetically modified vertebrates, e.g. transgenic
A01K 67/0278 - Knock-in vertebrates, e.g. humanised vertebrates
C07K 14/74 - Major histocompatibility complex [MHC]
C07K 16/00 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies
C07K 16/06 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies from serum
C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
A non-human animal is genetically modified with sequence encoding a human or humanized MHC molecule or associated molecule, e.g., β2 microglobulin, and expression of the sequence by the non-human animal induces tolerance to the corresponding human HLA from which the human or human MHC molecule is derived. The tolerance exhibited by these non-human animals allows these animals to generate specific antibody responses to the corresponding human HLA when such HLA is presenting a peptide that is antigenic to the non-human animal. Such an antibody response, which specifically targets a pMHC complex of interest without binding to the MHC molecule may be useful in immunotherapeutic modalities that target a component of the immunological synapse which provides the specificity of that interaction.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A01K 67/0275 - Genetically modified vertebrates, e.g. transgenic
A01K 67/0278 - Knock-in vertebrates, e.g. humanised vertebrates
C07K 14/74 - Major histocompatibility complex [MHC]
C07K 16/00 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies
C07K 16/06 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies from serum
C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
The present invention provides methods for identifying, quantifying, and/or characterizing at least one host cell protein (HCP) impurity in a sample containing AAV vectors. The HCP impurities can be enriched through a differential digestion, maintaining intact capsids while exposing the sample to mild denaturation. The mildly denatured sample can subsequently be subjected to enzymatic digestion, generating peptides which can be identified and quantified by liquid chromatography-mass spectrometry (LC-MS) analysis to identify, quantify and/or characterize said at least one HCP impurity.
In one instance, disclosed herein is an auto-injector, the auto-injector including: a housing; a container disposed within the housing, the container configured to store a medicament; a canister disposed within the housing, the canister configured to release a pressurized medium to expel the medicament from the container; a shroud coupled to the housing, the shroud configured to move between a first position and a second position relative to the housing to cause the pressurized medium to be released by the canister; and a mandrel assembly disposed within the housing, the mandrel assembly configured to urge the shroud outward relative to the housing toward the first position after the pressurized medium is released by the canister.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
96.
EXTENDED, HIGH DOSE VEGF ANTAGONIST REGIMENS FOR TREATMENT OF ANGIOGENIC EYE DISORDERS
The present invention relates to regimens for the treatment of angiogenic eye disorders such as nAMD, DR and DME characterized by high doses of aflibercept extended periods between doses.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
Guide RNAs and CRISPR/Cas systems targeting a C5 locus or gene, lipid nanoparticles or viral vectors comprising such guide RNAs or CRISPR/Cas systems, and cells or animals comprising such guide RNAs or systems are provided. Methods of modifying or knocking down or knocking out a C5 locus or gene using the CRISPR/Cas systems are also provided, as well as use of the CRISPR/Cas systems in prophylactic and therapeutic applications for treatment and/or prevention of a disease, disorder, or condition associated with C5 and/or for ameliorating at least one symptom associated with such disease, disorder, or condition.
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
An auto-injector, including a housing and a cartridge disposed within the housing, the cartridge enclosing a medicament. The auto-injector further includes a fluid conduit configured to deliver the medicament from the cartridge to a patient and movable from a retracted configuration to a deployed configuration associated with administering the medicament, and a temperature sensor configured to determine a temperature of the medicament. A controller the temperature sensor is configured to: send a first signal to an external device when the temperature sensor senses that a temperature of the medicament rises above a threshold temperature.
A packaging comprising a tray having a body surrounding an exposed cavity and a groove; a lip surrounding the opening, wherein the lip extends radially outward from the cavity and defines a periphery of the tray; a retainer for covering a portion of the opening of the tray, the retainer having an aperture, wherein the aperture corresponds to the groove of the tray; a removable cover; and an auto-injector contained within the cavity, wherein the auto-injector is pre-filed with a medicament or other fluid.
A61M 5/00 - Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular wayAccessories therefor, e.g. filling or cleaning devices, arm rests
