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CL2020002043A1 - Métodos y composiciones para la entrega de proteínas terapeuticas. - Google Patents

Métodos y composiciones para la entrega de proteínas terapeuticas.

Info

Publication number
CL2020002043A1
CL2020002043A1 CL2020002043A CL2020002043A CL2020002043A1 CL 2020002043 A1 CL2020002043 A1 CL 2020002043A1 CL 2020002043 A CL2020002043 A CL 2020002043A CL 2020002043 A CL2020002043 A CL 2020002043A CL 2020002043 A1 CL2020002043 A1 CL 2020002043A1
Authority
CL
Chile
Prior art keywords
delivery
compositions
methods
therapeutic proteins
proteins
Prior art date
Application number
CL2020002043A
Other languages
English (en)
Inventor
Andrew Baik
Katherine Cygnar
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 CL2020002043A1 publication Critical patent/CL2020002043A1/es

Links

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405Glucanases
    • C12N9/2408Glucanases acting on alpha -1,4-glucosidic bonds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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
    • A61K47/6801Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • A61K47/6815Enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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
    • A61K47/6835Medicinal 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
    • A61K47/6849Medicinal 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 targeting a receptor, a cell surface antigen or a cell surface determinant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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
    • A61K47/6835Medicinal 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
    • A61K47/6875Medicinal 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
    • A61K47/6879Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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
    • A61K47/6835Medicinal 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
    • A61K47/6881Cluster-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/69Medicinal 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
    • A61K47/6901Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2839Immunoglobulins [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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2896Immunoglobulins [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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00Modified animals
    • A01K2207/15Humanized animals
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/072Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/15Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/505Medicinal preparations containing antigens or antibodies comprising antibodies
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Inorganic Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CL2020002043A 2018-02-07 2020-08-06 Métodos y composiciones para la entrega de proteínas terapeuticas. CL2020002043A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862627721P 2018-02-07 2018-02-07
US201862777683P 2018-12-10 2018-12-10

Publications (1)

Publication Number Publication Date
CL2020002043A1 true CL2020002043A1 (es) 2021-07-23

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Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020002043A CL2020002043A1 (es) 2018-02-07 2020-08-06 Métodos y composiciones para la entrega de proteínas terapeuticas.

Country Status (16)

Country Link
US (2) US12258597B2 (es)
EP (1) EP3749373A1 (es)
JP (3) JP7779653B2 (es)
KR (1) KR20200118151A (es)
CN (1) CN112040985A (es)
AU (2) AU2019218892B2 (es)
BR (1) BR112020016005A2 (es)
CA (1) CA3090519A1 (es)
CL (1) CL2020002043A1 (es)
CO (1) CO2020011068A2 (es)
IL (2) IL322464A (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 (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MY189159A (en) 2015-07-06 2022-01-29 Regeneron Pharma Multispecific antigen-binding molecules and uses thereof
US20190112588A1 (en) * 2015-12-08 2019-04-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
MY198804A (en) 2017-06-07 2023-09-28 Regeneron Pharma Compositions 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
BR112020016005A2 (pt) 2018-02-07 2020-12-15 Regeneron Pharmaceuticals, Inc. Método de administração de uma proteína terapêutica ao sistema nervoso central, proteína terapêutica multidomínio, polinucleotídeo, vetor de terapia gênica, e, uso de um nucleotídeo que codifica a proteína terapêutica multidomínio
CN112135624B (zh) * 2018-05-17 2024-07-19 瑞泽恩制药公司 抗cd63抗体、缀合物和其用途
WO2020005341A1 (en) * 2018-06-29 2020-01-02 Icahn School Of Medicine At Mount Sinai Anc80 encoding sphingolipid-metabolizing proteins
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
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
EA202190416A1 (ru) 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. Мышечно-специфические комлексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
AU2020388634A1 (en) * 2019-11-19 2022-06-23 AskBio 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
CN116981774A (zh) * 2021-01-12 2023-10-31 Jcr制药股份有限公司 整合有编码配体与具有生理活性的蛋白质的融合蛋白的基因的核酸分子
US20250082640A1 (en) * 2021-04-23 2025-03-13 University Of Cincinnati Therapeutics for Hyponatremia and Polycystic Kidney Disease
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
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
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス,インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
PE20241212A1 (es) * 2021-11-04 2024-06-06 Regeneron Pharma Particulas virales redirigidas a musculo esqueletico
TW202334428A (zh) * 2021-12-28 2023-09-01 日商Jcr製藥股份有限公司 用於安全的基因治療之抗轉鐵蛋白受體抗體與具有生理活性的蛋白質之融合蛋白質
WO2023133579A1 (en) 2022-01-10 2023-07-13 Regeneron Pharmaceuticals, Inc. Bbb-targeted gaa delivered as gene therapy treats cns and muscle in pompe disease model mice
WO2023150623A2 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
CN119301262A (zh) * 2022-04-01 2025-01-10 武田药品工业株式会社 用于具有cns表现的疾病的基因疗法
CN119183457A (zh) 2022-04-15 2024-12-24 达因疗法公司 用于治疗强直性肌营养不良的肌肉靶向复合物和制剂
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
KR20250134733A (ko) * 2022-12-16 2025-09-11 리제너론 파아마슈티컬스, 인크. Aav 입자에 결합하는 항원-결합 분자 및 용도
KR20250150011A (ko) * 2023-02-13 2025-10-17 리제너론 파마슈티칼스 인코포레이티드 항-인간 cacng1 항체를 이용한 근육 관련 장애 치료
US20250049896A1 (en) * 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
AR133385A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Inserción anti-tfr:gaa y anti-cd63:gaa para el tratamiento de la enfermedad de pompe
WO2025106529A2 (en) * 2023-11-14 2025-05-22 Cavalry Biosciences, Inc. Compositions for targeting muscle cells and uses thereof
CN117567648B (zh) * 2023-11-24 2025-10-28 北京大学 用于蛋白质细胞内递送的模块化融合蛋白
US20250242056A1 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
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 (183)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US543153A (en) 1895-07-23 kidder
US4444878A (en) 1981-12-21 1984-04-24 Boston Biomedical Research Institute, Inc. Bispecific antibody determinants
US5229272A (en) 1989-04-25 1993-07-20 Igen, Inc. Catalytic antibody components
US5156965A (en) 1983-11-29 1992-10-20 Igen, Inc. Catalytic antibodies
US5126258A (en) 1984-09-07 1992-06-30 Scripps Clinic And Research Foundation Molecules with antibody combining sites that exhibit catalytic properties
US5030717A (en) 1986-09-17 1991-07-09 Scripps Clinic And Research Foundation Antibodies which catalyze hydrolysis of ester bonds
US5436153A (en) 1992-12-02 1995-07-25 Sprecher; Cindy A. Human amyloid protein precursor homolog and Kunitz-type inhibitor
IL107831A0 (en) 1992-12-02 1994-05-30 Zymogenetics Inc Novel human amyloid protein precursor homolog and kunitz-type inhibitor
CA2161114A1 (en) 1993-04-23 1994-11-10 Andrew D. Napper Catalytic antibodies which hydrolyze primary amides and methods for eliciting such antibodies
US5602021A (en) 1994-12-29 1997-02-11 Catalytic Antibodies, Inc. Method for generating proteolytic enzymes specific against a selected peptide sequence
US5585108A (en) 1994-12-30 1996-12-17 Nanosystems L.L.C. Formulations of oral gastrointestinal therapeutic agents in combination with pharmaceutically acceptable clays
US5731168A (en) 1995-03-01 1998-03-24 Genentech, Inc. Method for making heteromultimeric polypeptides
EP0840797B1 (en) 1995-07-25 2014-09-03 Crucell Holland 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
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
DE69719529T2 (de) 1996-10-17 2003-12-11 Immunomedics, Inc. Nichtantigenes toxinkonjugat und fusionsprotein eines internalisierendes rezeptorsystems
EP0989864A1 (en) 1997-06-11 2000-04-05 The School Of Pharmacy, University Of London Pharmaceutical compositions containing antibody-enzyme conjugates in combination with prodrugs
IT1293511B1 (it) 1997-07-30 1999-03-01 Gentili Ist Spa Anticorpi monoclonali catalitici ad attivita' proteasica per la lisi selettiva della componente proteica di placche e aggregati correlati
US6703488B1 (en) 1998-01-15 2004-03-09 Center For Molecular Medicine And Immunology Antibody/receptor targeting moiety for enhanced delivery of armed ligand
US6855804B2 (en) 1998-03-23 2005-02-15 Board Of Regents, The University Of Texas System Covalently reactive transition state analogs and methods of use thereof
ES2396693T3 (es) 1998-04-02 2013-02-25 Xintela Ab Heterodímero de integrina y subunidad del mismo
WO2000022110A2 (en) 1998-10-09 2000-04-20 President And Fellows Of Harvard College Targeted proteolysis by recruitment to ubiquitin protein ligases
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
EP1322750A4 (en) 2000-09-08 2004-09-29 California Inst Of Techn PROTEOLYSIS TARGET DRUG TARGET MEDICINE
US6649138B2 (en) 2000-10-13 2003-11-18 Quantum Dot Corporation Surface-modified semiconductive and metallic nanoparticles having enhanced dispersibility in aqueous media
US7105348B2 (en) 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US7431923B2 (en) 2005-01-03 2008-10-07 Arius Research Inc. Cytotoxicity mediation of cells evidencing surface expression of CD63
US7534429B2 (en) 2000-11-29 2009-05-19 Hoffmann-La Roche Inc. Cytotoxicity mediation of cells evidencing surface expression of CD63
US7442777B2 (en) 2000-11-29 2008-10-28 Arius Research Inc. Cytotoxicity mediation of cells evidencing surface expression of CD63
US20060210474A1 (en) 2000-11-29 2006-09-21 Young David S Cytotoxicity mediation of cells evidencing surface expression of CD63
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
DE60237475D1 (de) 2001-12-21 2010-10-07 Alcon Inc Verwendung von synthetischen anorganischen nanoteilchen als träger für augenmedikamente
AU2003222568B2 (en) 2002-01-11 2009-05-07 Bioasis Technologies, Inc. Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
AU2003289716A1 (en) 2002-09-12 2004-04-30 Incyte Corporation Molecules for diagnostics and therapeutics
US20050142141A1 (en) 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
ES2371913T3 (es) 2003-01-22 2012-01-11 Duke University Constructos mejorados para expresar polipéptidos lisosomales.
SE0301087D0 (sv) 2003-04-14 2003-04-14 Cartela Ab New monoclonal antibody
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
EP3513814A1 (en) 2003-05-01 2019-07-24 Genzyme Corporation Gene therapy for neurometabolic disorders
ES2373658T3 (es) 2003-06-18 2012-02-07 Bayer Pharma Aktiengesellschaft Nuevas entidades biológicas y el uso de las mismas.
US7371539B2 (en) 2003-12-03 2008-05-13 President And Fellows Of Harvard College Targeted polypeptide degradation
AU2005211775B2 (en) 2004-02-06 2009-10-08 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
AU2005212435B2 (en) 2004-02-10 2010-09-09 Biomarin Pharmaceutical Inc. Acid alpha-glucosidase and fragments thereof
US20060078542A1 (en) 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
EP1732599A4 (en) 2004-03-25 2008-05-14 Centocor Inc USE OF EMMPRINE ANTAGONISTS FOR THE TREATMENT OF DISEASES RELATED TO EXCESSIVE ANGIOGENESIS
US20060171926A1 (en) 2004-04-30 2006-08-03 Passini Marco A Gene therapy for neurometabolic disorders
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
JP5137814B2 (ja) 2005-04-06 2013-02-06 ジェンザイム・コーポレーション 治療ターゲティングのための酸不安定リンカーを介するpegおよびポリシアルリソソーム酵素のコンジュゲート
PL3058959T3 (pl) 2005-05-02 2019-07-31 Genzyme Corporation Terapia genowa dla zaburzeń neurometabolicznych
MX2007016039A (es) 2005-06-17 2008-10-27 Univ North Carolina Metodos, sistemas y materiales de fabricacion de nanoparticulas.
EP4174179B1 (en) 2005-08-23 2025-05-07 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
KR101410521B1 (ko) 2005-12-16 2014-06-20 아이비씨 파마슈티컬스, 인코퍼레이티드 다가 면역글로불린-계 생동성 어셈블리들
CA2641070C (en) 2006-02-06 2017-01-03 Ramot At Tel-Aviv University Ltd. Enzyme replacement therapy for treating lysosomal storage diseases
MY159787A (en) 2006-06-02 2017-01-31 Regeneron Pharma High affinity antibodies to human il-6 receptor
US20080089891A1 (en) 2006-07-26 2008-04-17 Arius Research, Inc. Cancerous disease modifying antibodies
GB0615662D0 (en) 2006-08-07 2006-09-13 Affitech As Antibody
HRP20140081T1 (hr) 2006-08-18 2014-03-28 Novartis Ag Prlr-specifiäśna antitijela i njihova uporaba
CA2669347A1 (en) 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
AU2008218109A1 (en) 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
ES2549121T3 (es) 2007-05-18 2015-10-23 Tokyo Metropolitan Institute Of Medical Science Composición farmacéutica para terapia de reemplazo enzimático
WO2009018122A2 (en) 2007-07-27 2009-02-05 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the cns
WO2009075815A1 (en) 2007-12-07 2009-06-18 Duke University Immunomodulating gene therapy
WO2009094561A1 (en) 2008-01-24 2009-07-30 The Government Of The United States Of America As Represented By The Secretary Of The Departmentof Induced internalization of surface receptors
EP3586868B1 (en) 2008-04-22 2023-11-08 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
CA2737519A1 (en) 2008-09-29 2010-04-01 Centocor Ortho Biotech Inc. Anti-cd147 antibodies, methods, and uses
EP4083072A1 (en) * 2008-10-06 2022-11-02 Minerva Biotechnologies Corporation Muc1* antibodies
EP2241576A1 (en) 2009-04-17 2010-10-20 Trion Pharma Gmbh Use of trifunctional bispecific antibodies for the treatment of tumors associated with CD133+/EpCAM+ cancer stem cells
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
KR20120027055A (ko) 2009-06-26 2012-03-20 리제네론 파라마큐티칼스 인코포레이티드 천연 면역글로불린 포맷을 가지는 용이하게 분리된 이중특이성 항체
KR101498098B1 (ko) 2009-07-01 2015-03-03 케메트 일렉트로닉스 코포레이션 고전압 성능을 가지는 고캐패시턴스 다층레이어
EP3165234B1 (de) 2009-07-31 2019-04-03 ethris GmbH Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
WO2011029823A1 (en) 2009-09-09 2011-03-17 Novartis Ag Monoclonal antibody reactive with cd63 when expressed at the surface of degranulated mast cells
EP2332995A1 (en) 2009-12-10 2011-06-15 Bayer Schering Pharma Aktiengesellschaft Neutralizing prolactin receptor antibodies and their therapeutic use
US20110223176A1 (en) 2010-03-11 2011-09-15 Abbott Laboratories Basigin binding proteins
WO2011140279A1 (en) 2010-05-04 2011-11-10 Wayne State University Aav-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells
US8809282B2 (en) 2010-05-06 2014-08-19 Duke University Method of reducing titers of antibodies specific for a therapeutic agent
US9347050B2 (en) 2010-09-29 2016-05-24 Oxyrane Uk Limited Mannosidases capable of uncapping mannose-1-phospho-6-mannose linkages and demannosylating phosphorylated N-glycans and methods of facilitating mammalian cellular uptake of glycoproteins
US8679478B2 (en) 2010-10-04 2014-03-25 Duke University Methods of lysosomal storage disease therapy
US9782342B2 (en) 2010-10-11 2017-10-10 Wichita State University Composite magnetic nanoparticle drug delivery system
PL2643468T3 (pl) 2010-11-22 2018-11-30 Amicus Therapeutics, Inc. Nowe sekwencje sygnałowe do poprawy ekspresji białek i wydzielania ekombinowanych enzymów i innych białek
SI2646470T1 (sl) 2010-11-30 2017-05-31 F. Hoffmann-La Roche Ag Protitelesa proti receptorjem antitransferina in njihova uporaba za prenos terapevtskega enoverižnega variabilnega fragmenta (scfv) prek krvno-možganske pregrade
ES2946169T3 (es) 2011-02-25 2023-07-13 Regeneron Pharma Ratones ADAM6
DK2687597T3 (en) 2011-03-16 2019-02-25 Amano Enzyme Inc MODIFIED ALPHA-GLUCOSIDASE AND USES OF SAME
US20140212503A1 (en) 2011-03-17 2014-07-31 Hyukjin Lee Delivery system
PT2691529T (pt) 2011-03-31 2019-09-27 Univ Iowa Res Found Partícula de aav2 que compreende uma proteína do capsídeo de aav2 e um vetor que compreende um ácido nucleico que codifica uma tripeptidil peptidase 1 (tpp1) para utilização no tratamento da lipofuscinose ceroide infantil tardia (lincl) num mamífero não roedor por injeção intraventricular ou entrega icv
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
JP2014513952A (ja) 2011-04-22 2014-06-19 ジェンザイム・コーポレーション 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ
US20120283503A1 (en) 2011-04-29 2012-11-08 The Johns Hopkins University Nanoparticle loaded stem cells and their use in mri guided hyperthermia
WO2012166653A2 (en) 2011-05-27 2012-12-06 Callidus Biopharma, Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
EP2530089A1 (en) 2011-06-03 2012-12-05 Bayer Pharma Aktiengesellschaft Neutralising prolactin receptor antibody Mat3 and its therapeutical use
KR20140125351A (ko) 2011-11-23 2014-10-28 이제니카 바이오테라퓨틱스, 인크. 항-cd98 항체 및 이의 사용 방법
CA2857647C (en) 2011-12-02 2022-04-19 Armagen Technologies, Inc. Methods and compositions for increasing arylsulfatase a activity in the cns
BR112014015238B1 (pt) 2011-12-20 2022-11-16 Regeneron Pharmaceuticals, Inc Método ex vivo para preparar um anticorpo que se liga a um antígeno de interesse compreendendo identificar sequências de ácido nucleico a partir de linfócitos b de camundongo geneticamente modificado pela colocação de um gene adam6
SG10201704846VA (en) 2012-03-14 2017-07-28 Regeneron Pharma Multispecific antigen-binding molecules and uses thereof
BR112014031788A2 (pt) 2012-06-19 2017-06-27 Univ Florida composições e métodos para o tratamento de diabetes
AU2013289206B2 (en) 2012-07-11 2018-08-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
RS58928B1 (sr) 2012-08-29 2019-08-30 Hoffmann La Roche Transporter za krvno-moždanu barijeru
US8883979B2 (en) 2012-08-31 2014-11-11 Bayer Healthcare Llc Anti-prolactin receptor antibody formulations
DK3115372T3 (da) 2012-11-27 2019-06-11 Biomarin Pharm Inc Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf
JP2016513114A (ja) 2013-02-20 2016-05-12 バレリオン セラピューティクス, エルエルシー フォーズス・コリ病の処置のための方法および組成物
WO2014130723A1 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of pompe disease
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
EP3622821A1 (en) 2013-05-15 2020-03-18 Regents of the University of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
WO2014185908A2 (en) 2013-05-15 2014-11-20 The Translational Genomics Research Institute Hybridoma clones, monoclonal antibodies, and methods of use
IL242088B2 (en) 2013-05-20 2023-12-01 Genentech Inc Anti-transferrin receptor antibodies and methods of use
US9388243B2 (en) 2013-05-29 2016-07-12 Samsung Electronics Co., Ltd. Method of target membrane protein depletion
US10906981B2 (en) 2013-07-19 2021-02-02 The Regents Of The University Of California Compositions and methods related to structures that cross the blood brain barrier
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
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
RU2693438C2 (ru) 2014-01-03 2019-07-02 Ф. Хоффманн-Ля Рош Аг Биспецифичные антитела против гаптена/против рецептора гематоэнцефалического барьера, их комплексы и их применение в качестве челноков гематоэнцефалического барьера
JP6557664B2 (ja) 2014-01-06 2019-08-07 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 一価の血液脳関門シャトルモジュール
US9629801B2 (en) 2014-01-10 2017-04-25 Wisconsin Alumni Research Foundation Blood-brain barrier targeting antibodies
PL3097197T3 (pl) 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
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
EP3148592A2 (en) 2014-06-02 2017-04-05 Regeneron Pharmaceuticals, Inc. Antibody-drug conjugates, their preparation and their therapeutic use
AU2015274647C1 (en) 2014-06-11 2020-01-30 Molecular Templates, Inc. Protease-cleavage resistant, Shiga toxin a subunit effector polypeptides and cell-targeted molecules comprising the same
WO2016044947A1 (en) 2014-09-26 2016-03-31 Exerkine Corporation Exosomes useful to treat lysosomal storage disease
EP3220958A1 (en) 2014-10-24 2017-09-27 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof
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
CN107207591A (zh) 2014-12-10 2017-09-26 豪夫迈·罗氏有限公司 血脑屏障受体抗体及使用方法
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
US10179817B2 (en) 2015-05-04 2019-01-15 Cytomx Therapeutics, Inc. Anti-CD71 antibodies, activatable anti-CD71 antibodies, and methods of use thereof
MY193078A (en) 2015-06-24 2022-09-26 Hoffmann La Roche Anti-transferrin receptor antibodies with tailored affinity
CN107849555B (zh) 2015-06-24 2022-01-04 Jcr制药股份有限公司 通过血脑屏障的抗人转铁蛋白受体抗体
MY189159A (en) 2015-07-06 2022-01-29 Regeneron Pharma Multispecific antigen-binding molecules and uses thereof
WO2017026497A1 (ja) 2015-08-10 2017-02-16 国立大学法人大阪大学 抗体
AU2016332900C1 (en) 2015-09-29 2024-07-04 Amgen Inc. ASGR inhibitors
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
CN108137697A (zh) 2015-10-02 2018-06-08 豪夫迈·罗氏有限公司 双特异性抗人cd20/人转铁蛋白受体抗体及使用方法
SG11201803218PA (en) 2015-10-23 2018-05-30 Univ Iowa Res Found Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
US20190112588A1 (en) * 2015-12-08 2019-04-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
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
MY198085A (en) 2015-12-30 2023-07-31 Amicus Therapeutics Inc Augmented acid alpha-glucosidase for the treatment of pompe disease
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
JP7676100B2 (ja) * 2016-02-05 2025-05-14 ジェンマブ アクティーゼルスカブ 改善された内部移行特性を有する多重特異性抗原結合分子
KR102618519B1 (ko) 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
EP3448891A1 (en) 2016-04-28 2019-03-06 Regeneron Pharmaceuticals, Inc. Methods of making multispecific antigen-binding molecules
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
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
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
CN116173232A (zh) 2016-06-08 2023-05-30 艾伯维公司 抗cd98抗体及抗体药物偶联物
CN109562168A (zh) 2016-06-08 2019-04-02 艾伯维公司 抗cd98抗体及抗体药物偶联物
US11097010B2 (en) 2016-08-06 2021-08-24 Ossianix, Inc. In vivo methods for selecting peptides that cross the blood brain barrier, related compositions and methods of use
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
AU2017322376B2 (en) 2016-09-12 2023-08-17 Association Institut De Myologie Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
JP2019537576A (ja) 2016-11-04 2019-12-26 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用
CN110382534B (zh) 2016-11-29 2023-05-09 里珍纳龙药品有限公司 治疗prlr阳性乳腺癌的方法
WO2018124121A1 (ja) 2016-12-26 2018-07-05 Jcrファーマ株式会社 血液脳関門を通過する新規な抗ヒトトランスフェリン受容体抗体
EP3919064A1 (en) 2017-01-27 2021-12-08 Xintela AB Prevention and treatment of bone and cartilage damage or disease
WO2018165619A1 (en) 2017-03-09 2018-09-13 Cytomx Therapeutics, Inc. Cd147 antibodies, activatable cd147 antibodies, and methods of making and use thereof
HRP20230576T1 (hr) 2017-05-15 2023-09-01 Amicus Therapeutics, Inc. Rekombinantna ljudska kisela alfa-glukozidaza
MX2019012381A (es) 2017-05-18 2020-01-23 Hoffmann La Roche Reduccion de reaccion secundaria relacionada con la aplicacion de un anticuerpo terapeutico.
MY198804A (en) * 2017-06-07 2023-09-28 Regeneron Pharma Compositions and methods for internalizing enzymes
JP2020527552A (ja) 2017-07-10 2020-09-10 バイエル・ファルマ・アクティエンゲゼルシャフト 男性型脱毛症および女性型脱毛症用のプロラクチン受容体抗体
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
EP3691692B8 (en) 2017-10-14 2021-05-26 AbbVie Inc. Anti-cd71 activatable antibody drug conjugates and methods of use thereof
EP3749756A4 (en) 2018-02-05 2021-12-08 Audentes Therapeutics, Inc. ELEMENTS OF TRANSCRIPTION REGULATION AND ASSOCIATED USES
CN111683972A (zh) 2018-02-05 2020-09-18 Jcr制药股份有限公司 用于将药剂递送至肌肉的方法
BR112020016005A2 (pt) 2018-02-07 2020-12-15 Regeneron Pharmaceuticals, Inc. Método de administração de uma proteína terapêutica ao sistema nervoso central, proteína terapêutica multidomínio, polinucleotídeo, vetor de terapia gênica, e, uso de um nucleotídeo que codifica a proteína terapêutica multidomínio
CA3093922A1 (en) 2018-03-15 2019-09-19 Valerion Therapeutics, Llc Methods and compositions for treatment of polyglucosan disorders
EP3552631A1 (en) 2018-04-10 2019-10-16 Inatherys Antibody-drug conjugates and their uses for the treatment of cancer
EP3794043A4 (en) 2018-05-16 2022-02-23 Spark Therapeutics, Inc. CODON-ENHANCED ACID ALPHA-GLUCOSIDASE EXPRESSION CASSETTES AND METHODS OF USE THEREOF
CN112135624B (zh) 2018-05-17 2024-07-19 瑞泽恩制药公司 抗cd63抗体、缀合物和其用途
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
WO2020028841A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating pompe disease
EP3840775A4 (en) 2018-08-24 2022-07-06 Spark Therapeutics, Inc. OPTIMIZED PROMOTER SEQUENCES, INTRON-FREE EXPRESSION CONSTRUCTS AND METHODS OF USE
AU2019381776A1 (en) 2018-11-16 2021-07-01 AskBio Inc. Therapeutic adeno-associated virus for treating Pompe disease
CA3121177A1 (en) 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
CA3124790A1 (en) 2019-01-09 2020-07-16 Vect-Horus Transferrin receptor-binding molecules, conjugates thereof and their uses
WO2020163480A1 (en) 2019-02-05 2020-08-13 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase and uses thereof
AU2020266829A1 (en) 2019-04-30 2021-11-11 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of Pompe disease
US20220331408A1 (en) 2019-07-09 2022-10-20 Genethon Treatment of glycogen storage disease (gsd)
WO2023133579A1 (en) 2022-01-10 2023-07-13 Regeneron Pharmaceuticals, Inc. Bbb-targeted gaa delivered as gene therapy treats cns and muscle in pompe disease model mice

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