CR20200282A - Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesis - Google Patents
Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesisInfo
- Publication number
- CR20200282A CR20200282A CR20200282A CR20200282A CR20200282A CR 20200282 A CR20200282 A CR 20200282A CR 20200282 A CR20200282 A CR 20200282A CR 20200282 A CR20200282 A CR 20200282A CR 20200282 A CR20200282 A CR 20200282A
- Authority
- CR
- Costa Rica
- Prior art keywords
- aav
- capsid
- virions
- associated virus
- adeno
- 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
- 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
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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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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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
- 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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/22—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- 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
- 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
- C12N2750/14145—Special targeting system for viral vectors
Abstract
<p>Se proveen proteínas variantes de la cápside de un virus adenoasociado (AAV) que tienen una o más modificaciones en la secuencia de aminoácidos en relación a una proteína de la cápside de AAV parental, que, cuando están presentes en un virión de AAV, confieren infectividad aumentada de uno o más tipos de células de la retina en comparación con la infectividad de las células de la retina por un virión de AAV que comprende la proteína no modificada de la cápside del AAV parental. También se proveen viriones de AAV recombinantes y composiciones farmacéuticas de los mismos que comprende una proteína variante de la cápside de AAV como se describe en la presente, métodos para elaborar estas proteínas de la cápside y viriones de rAAV, y métodos para usar estas proteínas de la cápside y viriones de rAAV en investigación y en la práctica clínica, por ejemplo en, por ejemplo, la administración de secuencias de ácido nucleico a una o más células de la retina para el tratamiento de trastornos y enfermedades de la retina.</p>
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762590976P | 2017-11-27 | 2017-11-27 | |
US201862664726P | 2018-04-30 | 2018-04-30 | |
PCT/US2018/062478 WO2019104279A1 (en) | 2017-11-27 | 2018-11-26 | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
Publications (1)
Publication Number | Publication Date |
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CR20200282A true CR20200282A (es) | 2021-02-18 |
Family
ID=66631726
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CR20200282A CR20200282A (es) | 2017-11-27 | 2018-11-26 | Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesis |
Country Status (21)
Country | Link |
---|---|
US (1) | US11766489B2 (es) |
EP (3) | EP3717636B1 (es) |
JP (3) | JP7184894B2 (es) |
KR (1) | KR20200088853A (es) |
CN (1) | CN111770999A (es) |
AU (2) | AU2018372235B9 (es) |
BR (1) | BR112020010674A2 (es) |
CA (1) | CA3083472A1 (es) |
CL (1) | CL2020001360A1 (es) |
CR (1) | CR20200282A (es) |
DK (1) | DK3717636T3 (es) |
ES (1) | ES2946747T3 (es) |
FI (1) | FI3717636T3 (es) |
IL (1) | IL274712A (es) |
MX (1) | MX2020005451A (es) |
PE (1) | PE20210915A1 (es) |
PH (1) | PH12020550706A1 (es) |
PT (1) | PT3717636T (es) |
SG (1) | SG11202004545XA (es) |
UA (1) | UA127831C2 (es) |
WO (1) | WO2019104279A1 (es) |
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BR112020012336A2 (pt) * | 2017-12-19 | 2021-03-30 | Akouos, Inc. | Administração de anticorpos terapêuticos mediada por aav para o ouvido interno |
US11149256B2 (en) | 2018-09-26 | 2021-10-19 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
US20220411508A1 (en) * | 2019-09-09 | 2022-12-29 | The Regents Of The University Of California | Compositions and methods for making and using multispecific antibodies |
EP4028531A2 (en) * | 2019-09-09 | 2022-07-20 | Massachusetts Eye and Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav |
CN114786731A (zh) | 2019-10-10 | 2022-07-22 | 科达制药股份有限公司 | 治疗眼部病症的方法 |
AU2020373326A1 (en) * | 2019-10-31 | 2022-06-02 | Medizinische Hochschule Hannover | AAV vector variants for ocular gene delivery |
US20240067678A1 (en) * | 2020-12-16 | 2024-02-29 | Children's Medical Research Institute | Adeno-associated virus capsids and vectors |
US20240059743A1 (en) * | 2020-12-16 | 2024-02-22 | Chidren's Medical Research Institute | Aav capsids and vectors |
CN113041360A (zh) * | 2021-04-01 | 2021-06-29 | 深圳廷美奥生物技术有限公司 | 一种用于治疗年龄相关性黄斑变性的药物 |
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CN113481237A (zh) * | 2021-06-29 | 2021-10-08 | 厦门朔望医药科技有限公司 | 一种预防和治疗新生血管眼部疾病的基因药物 |
AU2022340595A1 (en) * | 2021-09-06 | 2024-04-18 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Treatment of rpe65-associated eye diseases and disorders |
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WO2023089151A1 (en) * | 2021-11-19 | 2023-05-25 | The University Of Bristol | Materials and methods for treatment of macular degeneration |
CN116444626A (zh) * | 2021-12-28 | 2023-07-18 | 成都弘基生物科技有限公司 | 经过修饰的aav衣壳蛋白及其用途 |
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CN117180455A (zh) * | 2022-06-07 | 2023-12-08 | 成都弘基生物科技有限公司 | 腺相关病毒药物组合物及其用途 |
WO2024084075A1 (en) | 2022-10-20 | 2024-04-25 | Sparingvision | Compositions and methods for treating retinal degenerative disorders |
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