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
Links
Classifications
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- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2839—Immunoglobulins [IG], 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
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- 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
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- 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
- A61K47/51—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
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- 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
- A61K47/51—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
- A61K47/6849—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 targeting a receptor, a cell surface antigen or a cell surface determinant
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- 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
- A61K47/51—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
- A61K47/6875—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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- 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
- A61K47/51—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
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- 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
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- 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
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- 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
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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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2881—Immunoglobulins [IG], 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 [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2896—Immunoglobulins [IG], 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
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- 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)
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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)
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- 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
Landscapes
- 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)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Cell Biology (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Inorganic Chemistry (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)
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 |
Family
ID=65576681
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 (https=) |
| EP (1) | EP3749373A1 (https=) |
| JP (3) | JP7779653B2 (https=) |
| KR (1) | KR20200118151A (https=) |
| CN (1) | CN112040985A (https=) |
| AU (2) | AU2019218892B2 (https=) |
| BR (1) | BR112020016005A2 (https=) |
| CA (1) | CA3090519A1 (https=) |
| CL (1) | CL2020002043A1 (https=) |
| CO (1) | CO2020011068A2 (https=) |
| IL (2) | IL276464B2 (https=) |
| MA (1) | MA51796A (https=) |
| MX (1) | MX2020008274A (https=) |
| PH (1) | PH12020551180A1 (https=) |
| SG (1) | SG11202007363TA (https=) |
| WO (1) | WO2019157224A1 (https=) |
Families Citing this family (44)
| 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 |
| AU2016365834B2 (en) * | 2015-12-08 | 2023-09-28 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| FI3635009T3 (fi) | 2017-06-07 | 2026-04-07 | Regeneron Pharma | Koostumuksia ja menetelmiä entsyymien internalisoimiseksi |
| KR20200122320A (ko) | 2018-01-16 | 2020-10-27 | 씨엘에스 테라퓨틱스 리미티드 | 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료 |
| JP7779653B2 (ja) | 2018-02-07 | 2025-12-03 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 治療用タンパク質送達のための方法および組成物 |
| EP3793591A1 (en) * | 2018-05-17 | 2021-03-24 | 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 |
| 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | 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 |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| CA3159018A1 (en) * | 2019-11-19 | 2021-05-27 | Michael W. O'CALLAGHAN | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
| CA3203945A1 (en) * | 2021-01-08 | 2022-07-14 | Dmitry Dmitrievich Genkin | Combination of deoxyribonuclease enzyme and cell therapies for treatment of cancer |
| WO2022153957A1 (ja) * | 2021-01-12 | 2022-07-21 | Jcrファーマ株式会社 | リガンドと生理活性を有する蛋白質の融合蛋白質をコードする遺伝子が組み込まれた核酸分子 |
| WO2022226313A1 (en) * | 2021-04-23 | 2022-10-27 | University Of Cincinnati | Therapeutics for hyponatremia and polycystic kidney disease |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11672872B2 (en) | 2021-07-09 | 2023-06-13 | Dyne Therapeutics, Inc. | Anti-transferrin receptor antibody and uses thereof |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| IL309936A (en) | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of dystrophinopathy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| JP2024540181A (ja) * | 2021-11-04 | 2024-10-31 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 骨格筋に再標的化されたウイルス粒子 |
| 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 |
| 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 |
| EP4473103A2 (en) | 2022-02-02 | 2024-12-11 | 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 |
| US20250339553A1 (en) | 2022-04-15 | 2025-11-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
| AU2023316646A1 (en) | 2022-07-29 | 2024-12-12 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a modified transferrin receptor locus |
| AU2023367210A1 (en) * | 2022-10-27 | 2025-05-01 | California Institute Of Technology | Targets for receptor-mediated control of therapeutic biodistribution and efficacy |
| CN120712281A (zh) * | 2022-12-16 | 2025-09-26 | 瑞泽恩制药公司 | 结合到aav颗粒的抗原结合分子和用途 |
| KR20250150011A (ko) * | 2023-02-13 | 2025-10-17 | 리제너론 파마슈티칼스 인코포레이티드 | 항-인간 cacng1 항체를 이용한 근육 관련 장애 치료 |
| AU2024317483A1 (en) | 2023-07-28 | 2026-01-29 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease |
| US20250049896A1 (en) * | 2023-07-28 | 2025-02-13 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency |
| AU2024315073A1 (en) | 2023-07-28 | 2026-01-22 | Regeneron Pharmaceuticals, Inc. | Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion |
| 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 |
| WO2025160324A2 (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 |
| WO2026037757A1 (en) | 2024-08-13 | 2026-02-19 | F. Hoffmann-La Roche Ag | Modified aav particles |
| WO2026050618A1 (en) | 2024-08-29 | 2026-03-05 | Regeneron Pharmaceuticals, Inc. | Polynucleotide constructs and cell lines for recombinant aav production |
Family Cites Families (183)
| 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 |
| IL107831A0 (en) | 1992-12-02 | 1994-05-30 | Zymogenetics Inc | Novel human amyloid protein precursor homolog and kunitz-type inhibitor |
| US5436153A (en) | 1992-12-02 | 1995-07-25 | Sprecher; Cindy A. | Human amyloid protein precursor homolog and Kunitz-type inhibitor |
| EP0696318A4 (en) | 1993-04-23 | 1998-04-15 | Igen Inc | CATALYTIC ANTIBODIES HYDROLYZING PRIMARY AMIDES AND METHOD FOR DISPLAYING THESE 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 |
| JPH11510050A (ja) | 1995-07-25 | 1999-09-07 | イントロヘーネ ベスローテン フェンノートシャップ | 標的遺伝子送達のための方法および手段 |
| 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 |
| WO1998016254A1 (en) | 1996-10-17 | 1998-04-23 | Immunomedics, Inc. | Non-antigenic toxin-conjugate and fusion protein of internalizing receptor system |
| WO1998056425A1 (en) | 1997-06-11 | 1998-12-17 | 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 |
| CA2324381C (en) | 1998-04-02 | 2013-02-26 | Cartela Ab | An integrin heterodimer and a subunit thereof |
| US7223556B1 (en) | 1998-10-09 | 2007-05-29 | President And Fellows Of Harvard College | Targeted proteolysis by recruitment to ubiquitin protein ligases |
| WO2001097829A2 (en) | 2000-06-19 | 2001-12-27 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US20040204379A1 (en) | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US7041298B2 (en) | 2000-09-08 | 2006-05-09 | California Institute Of Technology | Proteolysis targeting chimeric pharmaceutical |
| 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 |
| US20060210474A1 (en) | 2000-11-29 | 2006-09-21 | Young David S | 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 |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| BR0215238A (pt) | 2001-12-21 | 2005-05-31 | Alcon Inc | Uso de nanopartìculas inor ânicas sintéticas como veìculos para drogas oftálmicas e óticas |
| 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 |
| ATE521701T1 (de) | 2003-01-22 | 2011-09-15 | Univ Duke | Verbesserte konstrukte zur expression lysosomaler polypeptide |
| SE0301087D0 (sv) | 2003-04-14 | 2003-04-14 | Cartela Ab | New monoclonal antibody |
| ES2562490T3 (es) | 2003-05-01 | 2016-03-04 | Genzyme Corporation | Terapia génica para trastornos neurometabólicos |
| US20040258666A1 (en) | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| JP2006527590A (ja) | 2003-06-18 | 2006-12-07 | ディレボ・ビオテク・アーゲー | 新規生物学的実体およびその使用 |
| US7371539B2 (en) | 2003-12-03 | 2008-05-13 | President And Fellows Of Harvard College | Targeted polypeptide degradation |
| CA2556245A1 (en) | 2004-02-06 | 2005-08-25 | Biomarin Pharmaceutical Inc. | Manufacture of highly phosphorylated lysosomal enzymes and uses thereof |
| WO2005077333A2 (en) | 2004-02-10 | 2005-08-25 | University Of Florida Research Foundation, Inc. | Gel-based delivery of recombinant adeno-associated virus vectors |
| CN1922313B (zh) | 2004-02-10 | 2011-10-26 | 生物马林医药公司 | 酸性α-糖苷酶及其片段 |
| AU2004317548A1 (en) | 2004-03-25 | 2005-10-06 | Centocor, Inc. | Use of EMMPRIN antagonists for the treatment of diseases associated with 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 |
| EP1877099B1 (en) | 2005-04-06 | 2012-09-19 | Genzyme Corporation | Therapeutic conjugates comprising a lysosomal enzyme, polysialic acid and a targeting moiety |
| EP1879624B1 (en) | 2005-05-02 | 2011-09-21 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| MX2007016039A (es) | 2005-06-17 | 2008-10-27 | Univ North Carolina | Metodos, sistemas y materiales de fabricacion de nanoparticulas. |
| EP3611266B1 (en) | 2005-08-23 | 2022-11-09 | The Trustees of the University of Pennsylvania | Rna containing modified nucleosides and methods of use thereof |
| CN101374546B (zh) | 2005-12-16 | 2016-03-23 | Ibc医药公司 | 基于免疫球蛋白的多价生物活性装配体 |
| EP1981546B1 (en) | 2006-02-06 | 2014-06-04 | The Medical Research and Infrastructure Fund of the Tel-Aviv Sourasky Medical Center | Enzyme replacement therapy for treating lysosomal storage diseases |
| ES2398076T3 (es) | 2006-06-02 | 2013-03-13 | Regeneron Pharmaceuticals, Inc. | Anticuerpos de alta afinidad contra el receptor de IL-6 humano |
| 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 |
| AR062435A1 (es) | 2006-08-18 | 2008-11-05 | Xoma Technology Ltd | Anticuerpo especifico prlr (receptor de prolactina) y sus usos |
| US20090117091A1 (en) | 2006-11-13 | 2009-05-07 | Lebowitz Jonathan | Methods for treating pompe disease |
| EP2132309A4 (en) | 2007-02-23 | 2011-01-05 | Univ Florida | COMPOSITIONS AND METHODS OF TREATING GLYCOLOGICAL MEMORY DISEASES |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2166093B1 (en) | 2007-05-18 | 2015-09-16 | Tokyo Metropolitan Institute of Medical Science | Pharmaceutical composition for enzyme replacement therapy |
| AU2008282496B2 (en) | 2007-07-27 | 2013-04-04 | 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 |
| EP2238162A1 (en) | 2008-01-24 | 2010-10-13 | The Government of the United States of America as represented by The Secretary of the Department of Health and Human Services | Induced internalization of surface receptors |
| EP4154903A1 (en) | 2008-04-22 | 2023-03-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| HRP20170698T1 (hr) | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| AU2009296937B2 (en) | 2008-09-29 | 2015-02-12 | Centocor Ortho Biotech Inc. | Anti-CD147 antibodies, methods, and uses |
| US20170204191A1 (en) * | 2008-10-06 | 2017-07-20 | Cynthia C. Bamdad | 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 |
| EP2475376B1 (en) | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| JP5816170B2 (ja) | 2009-06-26 | 2015-11-18 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. | 天然の免疫グロブリン形式を有する容易に単離される二重特異性抗体 |
| US8885319B2 (en) | 2009-07-01 | 2014-11-11 | Kemet Electronics Corporation | High capacitance multilayer with high voltage capability |
| 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" |
| 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 |
| WO2011139379A2 (en) | 2010-05-06 | 2011-11-10 | Duke University | A method of treating patients undergoing protein replacement therapy, gene replacement therapy, or other therapeutic modalities |
| 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 |
| US9279007B2 (en) | 2010-11-22 | 2016-03-08 | Amicus Therapeutics, Inc. | Signal sequences to improve protein expressions and secretion of recombinant enzymes and other proteins |
| AR084020A1 (es) | 2010-11-30 | 2013-04-17 | Genentech Inc | Anticuerpos para el receptor de la barrera hematoencefalica de baja afinidad y sus usos |
| RU2722373C2 (ru) | 2011-02-25 | 2020-05-29 | Редженерон Фармасьютикалс, Инк. | Мыши 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 |
| ES2745470T3 (es) | 2011-03-31 | 2020-03-02 | Univ Iowa Res Found | Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración 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 |
| MX2013012345A (es) | 2011-04-22 | 2015-05-07 | Genzyme Corp | Alfa glucosidasa acida modificada con procesamiento acelerado. |
| US20120283503A1 (en) | 2011-04-29 | 2012-11-08 | The Johns Hopkins University | Nanoparticle loaded stem cells and their use in mri guided hyperthermia |
| LT2714752T (lt) | 2011-05-27 | 2018-03-12 | Amicus Therapeutics, Inc. | Nukreipiančių peptidų kopuliavimo ant rekombinantinių lizosominių fermentų būdai, skirti lizosominio kaupimo ligų gydymo pagerinimui |
| EP2530089A1 (en) | 2011-06-03 | 2012-12-05 | Bayer Pharma Aktiengesellschaft | Neutralising prolactin receptor antibody Mat3 and its therapeutical use |
| JP2015501639A (ja) | 2011-11-23 | 2015-01-19 | アイジェニカ バイオセラピューティクス インコーポレイテッド | 抗cd98抗体およびその使用方法 |
| AU2012346448B2 (en) | 2011-12-02 | 2017-09-14 | Armagen, Inc. | Methods and compositions for increasing arylsulfatase A activity in the CNS |
| RU2664181C2 (ru) | 2011-12-20 | 2018-08-15 | Регенерон Фармасьютикалз, Инк. | Мыши с гуманизированной легкой цепью |
| MX361875B (es) | 2012-03-14 | 2018-12-18 | Regeneron Pharma | Moléculas de unión de antígeno multiespecifícas y usos de las mismas. |
| RS61055B1 (sr) | 2012-06-19 | 2020-12-31 | Univ Florida | Kompozicije i postupci za lečenje bolesti |
| DK3444342T3 (da) | 2012-07-11 | 2020-08-24 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme |
| RU2711552C2 (ru) | 2012-08-29 | 2020-01-17 | Ф. Хоффманн-Ля Рош Аг | Шаттл для гематоэнцефалического барьера |
| US8883979B2 (en) | 2012-08-31 | 2014-11-11 | Bayer Healthcare Llc | Anti-prolactin receptor antibody formulations |
| HUE039334T2 (hu) | 2012-11-27 | 2018-12-28 | Biomarin Pharm Inc | Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai |
| EP2981551B1 (en) | 2013-02-20 | 2020-06-03 | Valerion Therapeutics, LLC | Methods and compositions for treatment of pompe disease |
| WO2014130722A1 (en) | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of forbes-cori disease |
| EP2968538A1 (en) | 2013-03-15 | 2016-01-20 | Bayer HealthCare LLC | Anti-prolactin receptor antibody formulations |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| US9738717B2 (en) | 2013-05-15 | 2017-08-22 | The Translational Genomics Research Institute | Hybridoma clones, monoclonal antibodies, and methods of use |
| WO2014189973A2 (en) | 2013-05-20 | 2014-11-27 | Genentech, Inc. | Anti-transferrin receptor antibodies and methods of use |
| CN104211814A (zh) | 2013-05-29 | 2014-12-17 | 三星电子株式会社 | 用于消耗靶膜蛋白的组合物 |
| WO2015009961A1 (en) | 2013-07-19 | 2015-01-22 | Armagen Technologies, Inc. | 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 |
| CA2933384A1 (en) | 2014-01-03 | 2015-07-09 | F. Hoffmann-La Roche Ag | Bispecific anti-hapten/anti-blood brain barrier receptor antibodies, complexes thereof and their use as blood brain barrier shuttles |
| CA2932547C (en) | 2014-01-06 | 2023-05-23 | 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 |
| EP3097197B1 (en) | 2014-01-21 | 2020-12-16 | Vrije Universiteit Brussel | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| 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 |
| CA2950155C (en) | 2014-06-02 | 2021-11-23 | Regeneron Pharmaceuticals, Inc. | Biologically active molecule conjugates, reagents and methods of manufacture, and therapeutic uses |
| ES2919749T3 (es) | 2014-06-11 | 2022-07-28 | Molecular Templates Inc | Moléculas dirigidas a células citotóxicas resistentes a la escisión por proteasa |
| CA2962081A1 (en) | 2014-09-26 | 2016-03-31 | Exerkine Corporation | Exosomes useful to treat lysosomal storage disease |
| WO2016065319A1 (en) | 2014-10-24 | 2016-04-28 | 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 |
| EP3221361B1 (en) | 2014-11-19 | 2021-04-21 | Genentech, Inc. | Anti-transferrin receptor / anti-bace1 multispecific antibodies and methods of use |
| 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 |
| RU2017120039A (ru) | 2014-12-10 | 2019-01-10 | Дженентек, Инк. | Антитела к рецепторам гематоэнцефалического барьера и способы их применения |
| 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 |
| BR112017023862A2 (pt) | 2015-05-04 | 2018-07-17 | Cytomx Therapeutics Inc | anticorpos anti-cd71, anticorpos anti-cd71 ativáveis, e métodos de uso destes |
| TWI769982B (zh) | 2015-06-24 | 2022-07-11 | 日商Jcr製藥股份有限公司 | 通過血腦障壁之抗人類運鐵蛋白受體抗體 |
| MX391086B (es) | 2015-06-24 | 2025-03-21 | Hoffmann La Roche | Anticuerpos anti-receptor de transferrina con afinidad diseñada. |
| 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 |
| CA2997444A1 (en) | 2015-09-29 | 2017-04-06 | Amgen Inc. | Asgr inhibitors for reducing cholesterol levels |
| NZ741067A (en) | 2015-10-02 | 2023-07-28 | Hoffmann La Roche | Bispecific anti-human cd20/human transferrin receptor antibodies and methods of use |
| 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 |
| US20180236105A1 (en) | 2015-10-23 | 2018-08-23 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| AU2016365834B2 (en) | 2015-12-08 | 2023-09-28 | 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 |
| KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| AR107483A1 (es) | 2016-01-29 | 2018-05-02 | Hanmi Pharm Ind Co Ltd | Conjugado de enzimas terapéuticas |
| CN109071658A (zh) | 2016-02-05 | 2018-12-21 | 根马布股份公司 | 具有改进的内化特性的多特异性抗原-结合分子 |
| IL325155A (en) | 2016-03-30 | 2026-02-01 | Amicus Therapeutics Inc | Formulations containing recombinant acid alpha-glucosidase |
| WO2017190079A1 (en) | 2016-04-28 | 2017-11-02 | 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 |
| 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 |
| BR112018075630A2 (pt) | 2016-06-08 | 2019-03-19 | Abbvie Inc. | anticorpos anti-cd98 e conjugados de fármaco de anticorpo |
| BR112018075644A2 (pt) | 2016-06-08 | 2019-04-09 | Abbvie Inc. | anticorpos anti-cd98 e conjugados de anticorpo e fármaco |
| CA3033082A1 (en) | 2016-08-06 | 2018-02-15 | 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 |
| MX2019002842A (es) | 2016-09-12 | 2019-08-29 | Genethon | Variantes de alfa-glucosidasa acida y usos de las mismas. |
| EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3534892A4 (en) | 2016-11-04 | 2020-05-27 | The Children's Hospital of Philadelphia | GENE TRANSFER COMPOSITIONS, METHODS AND USE FOR TREATING NEURODEGENERATIVE DISEASES |
| WO2018102304A1 (en) | 2016-11-29 | 2018-06-07 | Regeneron Pharmaceuticals, Inc. | Methods of treating prlr positive breast cancer |
| TWI761413B (zh) | 2016-12-26 | 2022-04-21 | 日商Jcr製藥股份有限公司 | 通過血腦障壁之新穎抗人類運鐵蛋白受體抗體 |
| PT3573630T (pt) | 2017-01-27 | 2021-05-06 | Xintela Ab | Prevenção e tratamento de danos ou doenças na cartilagem e no osso |
| WO2018165619A1 (en) | 2017-03-09 | 2018-09-13 | Cytomx Therapeutics, Inc. | Cd147 antibodies, activatable cd147 antibodies, and methods of making and use thereof |
| EP3624831B1 (en) | 2017-05-15 | 2023-03-29 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| MX2019012381A (es) | 2017-05-18 | 2020-01-23 | Hoffmann La Roche | Reduccion de reaccion secundaria relacionada con la aplicacion de un anticuerpo terapeutico. |
| FI3635009T3 (fi) | 2017-06-07 | 2026-04-07 | Regeneron Pharma | Koostumuksia ja menetelmiä entsyymien internalisoimiseksi |
| PE20200481A1 (es) | 2017-07-10 | 2020-03-03 | Bayer Pharma AG | Anticuerpo del receptor de prolactina para la perdida de cabello con patrones masculinos y femeninos |
| SG11202002276VA (en) | 2017-09-20 | 2020-04-29 | 4D Molecular Therapeutics Inc | Adeno-associated virus variant capsids and methods of use thereof |
| RU2771292C2 (ru) | 2017-10-14 | 2022-04-29 | Эббви Инк. | Конъюгаты анти-cd71 активируемое антитело-лекарственное средство и способы их применения |
| AU2019215782A1 (en) | 2018-02-05 | 2020-07-16 | Jcr Pharmaceuticals Co., Ltd. | Method for delivering drug to muscle |
| US12296026B2 (en) | 2018-02-05 | 2025-05-13 | Astellas Gene Therapies, Inc. | Transcription regulatory elements and uses thereof |
| JP7779653B2 (ja) | 2018-02-07 | 2025-12-03 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 治療用タンパク質送達のための方法および組成物 |
| EP3765062A4 (en) | 2018-03-15 | 2022-04-13 | Valerion Therapeutics, LLC | METHODS AND COMPOSITIONS FOR THE TREATMENT OF POLYGLUCOUS DISORDERS |
| EP3552631A1 (en) | 2018-04-10 | 2019-10-16 | Inatherys | Antibody-drug conjugates and their uses for the treatment of cancer |
| CA3100213A1 (en) | 2018-05-16 | 2019-11-21 | Spark Therapeutics, Inc. | Codon-optimized acid .alpha.-glucosidase expression cassettes and methods of using same |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| 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 |
| JP2021533197A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | ポンペ病を処置するための筋標的化複合体およびそれらの使用 |
| US12290574B2 (en) | 2018-08-24 | 2025-05-06 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| WO2020102645A1 (en) | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| US12454701B2 (en) | 2018-12-05 | 2025-10-28 | 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 |
| CN114127275A (zh) | 2019-04-30 | 2022-03-01 | 宾夕法尼亚州大学信托人 | 可用于治疗庞贝病的组合物 |
| EP3996696A1 (en) | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| 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 |
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2019
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- 2019-02-07 EP EP19707943.7A patent/EP3749373A1/en active Pending
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- 2019-02-07 CA CA3090519A patent/CA3090519A1/en active Pending
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- 2019-02-07 MA MA051796A patent/MA51796A/fr unknown
- 2019-02-07 IL IL276464A patent/IL276464B2/en unknown
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- 2019-02-07 IL IL322464A patent/IL322464A/en unknown
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2020
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| RU2020129184A (ru) | 2022-03-09 |
| WO2019157224A1 (en) | 2019-08-15 |
| BR112020016005A2 (pt) | 2020-12-15 |
| US12258597B2 (en) | 2025-03-25 |
| JP2021512899A (ja) | 2021-05-20 |
| IL322464A (en) | 2025-09-01 |
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| CN112040985A (zh) | 2020-12-04 |
| JP7779653B2 (ja) | 2025-12-03 |
| AU2019218892B2 (en) | 2025-08-14 |
| CO2020011068A2 (es) | 2020-12-10 |
| JP7624469B2 (ja) | 2025-01-30 |
| KR20200118151A (ko) | 2020-10-14 |
| IL276464B1 (en) | 2025-09-01 |
| AU2025259791A1 (en) | 2025-11-20 |
| AU2019218892A1 (en) | 2020-10-01 |
| JP2025061494A (ja) | 2025-04-10 |
| MA51796A (fr) | 2020-12-16 |
| US20250197829A1 (en) | 2025-06-19 |
| MX2020008274A (es) | 2020-11-11 |
| SG11202007363TA (en) | 2020-08-28 |
| CA3090519A1 (en) | 2019-08-15 |
| US20200399623A1 (en) | 2020-12-24 |
| PH12020551180A1 (en) | 2021-06-07 |
| EP3749373A1 (en) | 2020-12-16 |
| IL276464B2 (en) | 2026-01-01 |
| JP2023054256A (ja) | 2023-04-13 |
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