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MX2020008274A - Metodos y composiciones para la administracion de proteinas terapeuticas. - Google Patents

Metodos y composiciones para la administracion de proteinas terapeuticas.

Info

Publication number
MX2020008274A
MX2020008274A MX2020008274A MX2020008274A MX2020008274A MX 2020008274 A MX2020008274 A MX 2020008274A MX 2020008274 A MX2020008274 A MX 2020008274A MX 2020008274 A MX2020008274 A MX 2020008274A MX 2020008274 A MX2020008274 A MX 2020008274A
Authority
MX
Mexico
Prior art keywords
therapeutic protein
compositions
methods
cns
protein delivery
Prior art date
Application number
MX2020008274A
Other languages
English (en)
Inventor
Katherine Cygnar
Andrew Baik
Maria Praggastis
Original Assignee
Regeneron Pharma
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of MX2020008274A publication Critical patent/MX2020008274A/es

Links

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • A61K47/6815—Enzymes
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
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    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
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    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6875—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody being a hybrid immunoglobulin
    • A61K47/6879—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody being a hybrid immunoglobulin the immunoglobulin having two or more different antigen-binding sites, e.g. bispecific or multispecific immunoglobulin
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6881—Cluster-antibody conjugates, i.e. the modifying agent consists of a plurality of antibodies covalently linked to each other or of different antigen-binding fragments covalently linked to each other
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2839—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the integrin superfamily
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2896—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against molecules with a "CD"-designation, not provided for elsewhere
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Inorganic Chemistry (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

Se describen composiciones y métodos para administrar una proteína terapéutica al sistema nervioso central (CNS), con el objetivo de tratar enfermedades y trastornos que afectan al CNS, tal como el tratamiento de enfermedades de almacenamiento lisosómico. Se proporcionan proteínas terapéuticas administradas a través de una cantidad terapéuticamente eficaz de una composición de nucleótido que codifica la proteína terapéutica conjugada a una proteína de unión a receptores de la superficie celular que cruza la barrera hematoencefálica (BBB).
MX2020008274A 2018-02-07 2019-02-07 Metodos y composiciones para la administracion de proteinas terapeuticas. MX2020008274A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862627721P 2018-02-07 2018-02-07
US201862777683P 2018-12-10 2018-12-10
PCT/US2019/017116 WO2019157224A1 (en) 2018-02-07 2019-02-07 Methods and compositions for therapeutic protein delivery

Publications (1)

Publication Number Publication Date
MX2020008274A true MX2020008274A (es) 2020-11-11

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MX2020008274A MX2020008274A (es) 2018-02-07 2019-02-07 Metodos y composiciones para la administracion de proteinas terapeuticas.

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Country Link
US (1) US20200399623A1 (es)
EP (1) EP3749373A1 (es)
JP (2) JP2021512899A (es)
KR (1) KR20200118151A (es)
CN (1) CN112040985A (es)
AU (1) AU2019218892A1 (es)
BR (1) BR112020016005A2 (es)
CA (1) CA3090519A1 (es)
CL (1) CL2020002043A1 (es)
CO (1) CO2020011068A2 (es)
IL (1) IL276464A (es)
MA (1) MA51796A (es)
MX (1) MX2020008274A (es)
PH (1) PH12020551180A1 (es)
SG (1) SG11202007363TA (es)
WO (1) WO2019157224A1 (es)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE060136T2 (hu) * 2015-12-08 2023-02-28 Regeneron Pharma Enzimek internalizálására szolgáló készítmények és módszerek
IL271193B1 (en) 2017-06-07 2024-09-01 Regeneron Pharma Preparations and methods for internalizing enzymes
KR20200122320A (ko) 2018-01-16 2020-10-27 씨엘에스 테라퓨틱스 리미티드 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료
US20210079109A1 (en) * 2018-05-17 2021-03-18 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
EP3813871A4 (en) * 2018-06-29 2022-10-12 Icahn School of Medicine at Mount Sinai SPHINGOLIPID METABOLIZING PROTEINS ENCODING FOR ANC80
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20230038520A1 (en) * 2019-11-19 2023-02-09 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders
JP2024504926A (ja) * 2021-01-08 2024-02-02 シーエルエス セラピューティクス リミテッド がんの処置のためのデオキシリボヌクレアーゼ酵素と細胞療法の組み合わせ
WO2022226313A1 (en) * 2021-04-23 2022-10-27 University Of Cincinnati Therapeutics for hyponatremia and polycystic kidney disease
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
IL312598A (en) * 2021-11-04 2024-07-01 Regeneron Pharma Viral particles are retargeted to skeletal muscle
TW202346348A (zh) 2022-01-10 2023-12-01 美商再生元醫藥公司 作為基因治療所遞送之bbb靶向的gaa在龐貝氏症小鼠模型中治療cns及肌肉
AU2023216255A1 (en) 2022-02-02 2024-07-11 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
WO2023187728A1 (en) * 2022-04-01 2023-10-05 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
AU2023254846A1 (en) 2022-04-15 2024-10-10 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
WO2024026488A2 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a modified transferrin receptor locus
WO2024092164A1 (en) * 2022-10-27 2024-05-02 California Institute Of Technology Targets for receptor-mediated control of therapeutic biodistribution and efficacy
WO2024130175A2 (en) * 2022-12-16 2024-06-20 Regeneron Pharmaceuticals, Inc. Antigen-binding molecules that bind to aav particles and uses
WO2024173248A1 (en) * 2023-02-13 2024-08-22 Regeneron Pharmaceuticals, Inc. Treatment of muscle related disorders with anti-human cacng1 antibodies
CN118005798B (zh) * 2024-04-08 2024-06-28 北京大学第三医院（北京大学第三临床医学院） 一种抗维生素k2抗体及其应用

Family Cites Families (64)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US543153A (en) 1895-07-23 kidder
AU5684594A (en) 1992-12-02 1994-06-22 Novo Nordisk A/S Novel human amyloid protein precursor homologue and kunitz-type inhibitors
US5436153A (en) 1992-12-02 1995-07-25 Sprecher; Cindy A. Human amyloid protein precursor homolog and Kunitz-type inhibitor
US5585108A (en) 1994-12-30 1996-12-17 Nanosystems L.L.C. Formulations of oral gastrointestinal therapeutic agents in combination with pharmaceutically acceptable clays
WO1997005266A1 (en) 1995-07-25 1997-02-13 Introgene B.V. Methods and means for targeted gene delivery
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
DK1068238T3 (da) 1998-04-02 2013-01-14 Xintela Ab Integrinheterodimer og subunit deraf
US6649138B2 (en) 2000-10-13 2003-11-18 Quantum Dot Corporation Surface-modified semiconductive and metallic nanoparticles having enhanced dispersibility in aqueous media
CA2467763C (en) 2001-12-21 2011-09-13 Alcon, Inc. Use of synthetic inorganic nanoparticles as carriers for ophthalmic and otic drugs
SE0301087D0 (sv) 2003-04-14 2003-04-14 Cartela Ab New monoclonal antibody
EP1732599A4 (en) 2004-03-25 2008-05-14 Centocor Inc USE OF EMMPRINE ANTAGONISTS FOR THE TREATMENT OF DISEASES RELATED TO EXCESSIVE ANGIOGENESIS
EP1904932A4 (en) 2005-06-17 2013-02-27 Univ North Carolina METHODS, SYSTEMS AND MATERIALS FOR MANUFACTURING NANOPARTICLES
DK2578685T3 (da) 2005-08-23 2019-06-03 Univ Pennsylvania Rna indeholende modificerede nukleosider og fremgangsmåder til anvendelse deraf
GB0615662D0 (en) 2006-08-07 2006-09-13 Affitech As Antibody
AU2008282496B2 (en) 2007-07-27 2013-04-04 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the CNS
AU2009296937B2 (en) 2008-09-29 2015-02-12 Centocor Ortho Biotech Inc. Anti-CD147 antibodies, methods, and uses
KR101498098B1 (ko) 2009-07-01 2015-03-03 케메트 일렉트로닉스 코포레이션 고전압 성능을 가지는 고캐패시턴스 다층레이어
BR112012002291A2 (pt) 2009-07-31 2016-11-29 Ethris Gmbh "polirribonucleotídeo com uma sequência que codifica uma proteína ou fragmento de proteína, implante, e, processo para a seleção de sequências de nucleotídeos"
EP2332995A1 (en) 2009-12-10 2011-06-15 Bayer Schering Pharma Aktiengesellschaft Neutralizing prolactin receptor antibodies and their therapeutic use
WO2011112566A2 (en) 2010-03-11 2011-09-15 Abbott Laboratories Basigin binding proteins
US9782342B2 (en) 2010-10-11 2017-10-10 Wichita State University Composite magnetic nanoparticle drug delivery system
KR102099462B1 (ko) 2010-11-30 2020-04-10 제넨테크, 인크. 저친화도 혈액-뇌 장벽 수용체 항체 및 그의 용도
WO2012125987A2 (en) 2011-03-17 2012-09-20 Massachusetts Institute Of Technology Delivery system
WO2012136519A1 (en) 2011-04-06 2012-10-11 Bayer Pharma Aktiengesellschaft Use of neutralizing prolactin receptor antibodies for the treatment and prevention of antiestrogen-resistant breast cancer
US20120283503A1 (en) 2011-04-29 2012-11-08 The Johns Hopkins University Nanoparticle loaded stem cells and their use in mri guided hyperthermia
EP2530089A1 (en) 2011-06-03 2012-12-05 Bayer Pharma Aktiengesellschaft Neutralising prolactin receptor antibody Mat3 and its therapeutical use
RU2014124530A (ru) 2011-11-23 2015-12-27 Идженика, Инк. Антитела к cd98 и способы их применения
ES2983576T3 (es) 2011-12-02 2024-10-23 Armagen Inc Métodos y composiciones para aumentar la actividad arilsulfatasa en el SNC
US8883979B2 (en) 2012-08-31 2014-11-11 Bayer Healthcare Llc Anti-prolactin receptor antibody formulations
CN105025925A (zh) 2013-03-15 2015-11-04 拜尔健康护理有限责任公司 抗-催乳素受体抗体制剂
US9738717B2 (en) 2013-05-15 2017-08-22 The Translational Genomics Research Institute Hybridoma clones, monoclonal antibodies, and methods of use
EP3594240B1 (en) 2013-05-20 2023-12-06 F. Hoffmann-La Roche AG Anti-transferrin receptor antibodies and methods of use
WO2015009961A1 (en) 2013-07-19 2015-01-22 Armagen Technologies, Inc. Compositions and methods related to structures that cross the blood brain barrier
US9545451B2 (en) 2013-08-21 2017-01-17 Regeneron Pharmaceuticals, Inc. Anti-PRLR antibodies and methods for killing PRLR-expressing cells
TWI641620B (zh) 2013-08-21 2018-11-21 再生元醫藥公司 抗－ｐｒｌｒ抗體及其用途
PL3089996T3 (pl) 2014-01-03 2021-12-13 F. Hoffmann-La Roche Ag Dwuswoiste przeciwciała przeciw haptenowi/przeciw receptorowi występującemu w barierze krew-mózg, ich kompleksy i ich zastosowanie jako przenośniki wahadłowe występujące w barierze krew-mózg
WO2015101588A1 (en) 2014-01-06 2015-07-09 F. Hoffmann-La Roche Ag Monovalent blood brain barrier shuttle modules
US9629801B2 (en) 2014-01-10 2017-04-25 Wisconsin Alumni Research Foundation Blood-brain barrier targeting antibodies
JP2017114763A (ja) 2014-03-26 2017-06-29 第一三共株式会社 抗ｃｄ９８抗体−薬物コンジュゲート
US9951141B2 (en) 2014-06-02 2018-04-24 Regeneron Pharmaceuticals, Inc. Antibody-drug conjugates, their preparation and their therapeutic use
WO2016077840A2 (en) * 2014-11-14 2016-05-19 Ossianix, Inc. TfR SELECTIVE BINDING COMPOUNDS AND RELATED METHODS
CN107250158B (zh) 2014-11-19 2022-03-25 基因泰克公司 抗转铁蛋白受体/抗bace1多特异性抗体和使用方法
EP3221362B1 (en) 2014-11-19 2019-07-24 F.Hoffmann-La Roche Ag Anti-transferrin receptor antibodies and methods of use
MA41022A (fr) * 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
KR20170085595A (ko) 2014-12-10 2017-07-24 제넨테크, 인크. 혈뇌 장벽 수용체 항체 및 사용 방법
US10538589B2 (en) 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
MY180297A (en) 2015-06-24 2020-11-27 Hoffmann La Roche Anti-transferrin receptor antibodies with tailored affinity
CN108138172B (zh) 2015-08-10 2021-07-27 国立大学法人大阪大学 抗体
EP4435105A2 (en) 2015-09-29 2024-09-25 Amgen Inc. Asgr inhibitors for reduzing cholesterol levels
AR106189A1 (es) 2015-10-02 2017-12-20 Hoffmann La Roche ANTICUERPOS BIESPECÍFICOS CONTRA EL A-b HUMANO Y EL RECEPTOR DE TRANSFERRINA HUMANO Y MÉTODOS DE USO
RU2753390C1 (ru) 2015-10-02 2021-08-13 Ф. Хоффманн-Ля Рош Аг Биспецифические антитела к человеческому cd20/человеческому рецептору трансферрина и способы их применения
HUE060136T2 (hu) * 2015-12-08 2023-02-28 Regeneron Pharma Enzimek internalizálására szolgáló készítmények és módszerek
US10233252B2 (en) 2015-12-21 2019-03-19 Wisconsin Alumni Research Foundation pH-dependent antibodies targeting the transferrin receptor and methods of use thereof to deliver a therapeutic agent
WO2017134197A1 (en) * 2016-02-05 2017-08-10 Genmab A/S Multispecific antigen-binding molecule with improved internalization characteristics
CN105820250B (zh) 2016-04-29 2019-04-30 中国人民解放军第四军医大学 一种抗basigin人源化抗体及其应用
JP2019524651A (ja) 2016-06-08 2019-09-05 アッヴィ・インコーポレイテッド 抗ｃｄ９８抗体及び抗体薬物コンジュゲート
WO2017214458A2 (en) 2016-06-08 2017-12-14 Abbvie Inc. Anti-cd98 antibodies and antibody drug conjugates
BR112018075651A2 (pt) 2016-06-08 2019-04-09 Abbvie Inc. anticorpos anti-cd98 e conjugados anticorpo fármaco
EA201900312A1 (ru) 2016-11-29 2019-12-30 Регенерон Фармасьютикалз, Инк. Способы лечения пролактин-рецептор положительного рака молочной железы
LT3573630T (lt) 2017-01-27 2021-05-25 Xintela Ab Kaulų ir kremzlių pažeidimų arba ligų prevencija ir gydymas
US20180303952A1 (en) 2017-03-09 2018-10-25 Cytomx Therapeutics, Inc. Cd147 antibodies, activatable cd147 antibodies, and methods of making and use thereof
WO2018210898A1 (en) 2017-05-18 2018-11-22 F. Hoffmann-La Roche Ag Reduction of application-related side reaction of a therapeutic antibody
IL271193B1 (en) * 2017-06-07 2024-09-01 Regeneron Pharma Preparations and methods for internalizing enzymes
CN110799545A (zh) 2017-07-10 2020-02-14 拜耳制药股份公司 用于男性和女性模式脱发的催乳素受体抗体

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