CN113966236A - 眼睛病状的基因疗法 - Google Patents
眼睛病状的基因疗法 Download PDFInfo
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- CN113966236A CN113966236A CN202080041009.0A CN202080041009A CN113966236A CN 113966236 A CN113966236 A CN 113966236A CN 202080041009 A CN202080041009 A CN 202080041009A CN 113966236 A CN113966236 A CN 113966236A
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- 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
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
- A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
- A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
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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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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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
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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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
- C07K16/22—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/53—DNA (RNA) vaccination
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- 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
- A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
- C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/40—Immunoglobulins specific features characterized by post-translational modification
- C07K2317/41—Glycosylation, sialylation, or fucosylation
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/515—Complete light chain, i.e. VL + CL
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/52—Constant or Fc region; Isotype
- C07K2317/522—CH1 domain
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/55—Fab or Fab'
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
- C07K2317/567—Framework region [FR]
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
- C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/04—Uses of viruses as vector in vivo
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Ophthalmology & Optometry (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Obesity (AREA)
- Mycology (AREA)
- Urology & Nephrology (AREA)
- Vascular Medicine (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202511459925.7A CN121371216A (zh) | 2019-04-03 | 2020-04-02 | 眼睛病状的基因疗法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962828949P | 2019-04-03 | 2019-04-03 | |
| US62/828,949 | 2019-04-03 | ||
| US201962856533P | 2019-06-03 | 2019-06-03 | |
| US62/856,533 | 2019-06-03 | ||
| US201962946158P | 2019-12-10 | 2019-12-10 | |
| US62/946,158 | 2019-12-10 | ||
| PCT/US2020/026356 WO2020206098A1 (en) | 2019-04-03 | 2020-04-02 | Gene therapy for eye pathologies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202511459925.7A Division CN121371216A (zh) | 2019-04-03 | 2020-04-02 | 眼睛病状的基因疗法 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN113966236A true CN113966236A (zh) | 2022-01-21 |
Family
ID=70480820
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202080041009.0A Pending CN113966236A (zh) | 2019-04-03 | 2020-04-02 | 眼睛病状的基因疗法 |
| CN202511459925.7A Pending CN121371216A (zh) | 2019-04-03 | 2020-04-02 | 眼睛病状的基因疗法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202511459925.7A Pending CN121371216A (zh) | 2019-04-03 | 2020-04-02 | 眼睛病状的基因疗法 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220143221A1 (https=) |
| EP (2) | EP4667578A3 (https=) |
| JP (2) | JP2022521851A (https=) |
| KR (2) | KR20220004987A (https=) |
| CN (2) | CN113966236A (https=) |
| AU (1) | AU2020253462A1 (https=) |
| BR (1) | BR112021019825A2 (https=) |
| CA (1) | CA3135843A1 (https=) |
| IL (1) | IL286825A (https=) |
| MX (1) | MX2021011889A (https=) |
| SG (1) | SG11202110789VA (https=) |
| TW (1) | TW202102679A (https=) |
| WO (1) | WO2020206098A1 (https=) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115137553A (zh) * | 2022-07-11 | 2022-10-04 | 上海市同济医院 | 一种免造孔视网膜下液排出装置 |
| CN116965768A (zh) * | 2023-07-07 | 2023-10-31 | 中山大学中山眼科中心 | 一种自动定量分析眼内前房炎症程度的系统 |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201808426XA (en) | 2016-04-15 | 2018-10-30 | Univ Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| EP3727468A4 (en) | 2017-12-19 | 2021-09-22 | Akouos, Inc. | AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR |
| US12558434B2 (en) | 2018-02-20 | 2026-02-24 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| CA3136119A1 (en) * | 2019-04-10 | 2020-10-15 | University Of Utah Research Foundation | Htra1 modulation for treatment of amd |
| EP3752524A4 (en) * | 2019-04-27 | 2021-11-24 | Ocugen, Inc. | ADENO-ASSOCIATED VIRUS VECTOR MEDIATED GENE THERAPY FOR OPHTHALMIC DISEASE |
| US20230277685A1 (en) * | 2020-04-08 | 2023-09-07 | President And Fellows Of Harvard College | TGFß THERAPY FOR OCULAR AND NEURODEGENERATIVE DISEASES |
| CN111826386B (zh) * | 2020-07-30 | 2022-02-01 | 西南大学 | 一种调控棉花纤维呈色的融合基因及其表达载体和应用 |
| AU2021358781A1 (en) * | 2020-10-07 | 2023-05-18 | Tern Therapeutics, Llc | Gene therapy for ocular manifestations of cln2 disease |
| IL303317A (en) | 2020-12-01 | 2023-07-01 | Akouos Inc | ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA |
| CN116964209A (zh) * | 2021-02-12 | 2023-10-27 | 格雷菲克斯公司 | 视网膜疾病的基因治疗 |
| US20240316219A1 (en) * | 2021-05-03 | 2024-09-26 | North Carolina State University | Compositions and methods related to the treatment of ocular diseases in equines |
| JP2024525183A (ja) * | 2021-06-18 | 2024-07-10 | イカロベック リミテッド | 網膜障害 |
| JP2025503637A (ja) * | 2022-01-09 | 2025-02-04 | クリヤ セラピューティクス, インコーポレイテッド | 治療用抗igf-1r抗体をコードする核酸の送達のためのベクター構築物およびそれを使用する方法 |
| JPWO2024048688A1 (https=) * | 2022-08-31 | 2024-03-07 | ||
| IL319873A (en) | 2022-09-30 | 2025-05-01 | Regenxbio Inc | Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB |
| WO2026011181A1 (en) * | 2024-07-05 | 2026-01-08 | Wisconsin Alumni Research Foundation | Design and application of novel photoreceptor-specific promoters for retinal gene therapy applications |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
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| WO2016134375A1 (en) * | 2015-02-20 | 2016-08-25 | University Of Iowa Research Foundation | Methods and compositions for treating genetic eye diseases |
| WO2017072515A1 (en) * | 2015-10-28 | 2017-05-04 | Syncona Management Llp | Gene therapy |
| EP3452103A1 (en) * | 2016-04-15 | 2019-03-13 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
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2020
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| WO2016134375A1 (en) * | 2015-02-20 | 2016-08-25 | University Of Iowa Research Foundation | Methods and compositions for treating genetic eye diseases |
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| CN115137553A (zh) * | 2022-07-11 | 2022-10-04 | 上海市同济医院 | 一种免造孔视网膜下液排出装置 |
| CN116965768A (zh) * | 2023-07-07 | 2023-10-31 | 中山大学中山眼科中心 | 一种自动定量分析眼内前房炎症程度的系统 |
| CN116965768B (zh) * | 2023-07-07 | 2024-01-19 | 中山大学中山眼科中心 | 一种自动定量分析眼内前房炎症程度的系统 |
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| US20220143221A1 (en) | 2022-05-12 |
| EP4667578A3 (en) | 2026-04-01 |
| TW202102679A (zh) | 2021-01-16 |
| KR20260033621A (ko) | 2026-03-10 |
| CN121371216A (zh) | 2026-01-23 |
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| BR112021019825A2 (pt) | 2021-12-21 |
| AU2020253462A1 (en) | 2021-10-28 |
| EP4667578A2 (en) | 2025-12-24 |
| SG11202110789VA (en) | 2021-10-28 |
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| WO2020206098A9 (en) | 2021-02-04 |
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| MX2021011889A (es) | 2021-10-26 |
| IL286825A (en) | 2021-10-31 |
| CA3135843A1 (en) | 2020-10-08 |
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