PE20201264A1 - Variantes de capsides de virus adenoasociados y metodos de uso de estas - Google Patents
Variantes de capsides de virus adenoasociados y metodos de uso de estasInfo
- Publication number
- PE20201264A1 PE20201264A1 PE2020000407A PE2020000407A PE20201264A1 PE 20201264 A1 PE20201264 A1 PE 20201264A1 PE 2020000407 A PE2020000407 A PE 2020000407A PE 2020000407 A PE2020000407 A PE 2020000407A PE 20201264 A1 PE20201264 A1 PE 20201264A1
- Authority
- PE
- Peru
- Prior art keywords
- aav
- capsid
- methods
- virions
- adeno
- Prior art date
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- 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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- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- A61K38/1719—Muscle proteins, e.g. myosin or actin
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- 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
- A61K38/44—Oxidoreductases (1)
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- A—HUMAN NECESSITIES
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- 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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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- 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
- C07K14/01—DNA viruses
- C07K14/03—Herpetoviridae, e.g. pseudorabies virus
- C07K14/05—Epstein-Barr virus
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- 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
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- 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
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- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1048—Glycosyltransferases (2.4)
- C12N9/1051—Hexosyltransferases (2.4.1)
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- C12Y116/00—Oxidoreductases oxidizing metal ions (1.16)
- C12Y116/03—Oxidoreductases oxidizing metal ions (1.16) with oxygen as acceptor (1.16.3)
- C12Y116/03001—Ferroxidase (1.16.3.1), i.e. ceruloplasmin
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- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/01—Hexosyltransferases (2.4.1)
- C12Y204/01001—Phosphorylase (2.4.1.1)
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- 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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- 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)
- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
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- A—HUMAN NECESSITIES
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- A61K9/0012—Galenical forms characterised by the site of application
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- 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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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
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- 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
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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- Engineering & Computer Science (AREA)
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Abstract
En la presente se proporcionan proteinas de capsides variantes de virus adenoasociados (AAV) que tienen una o mas modificaciones en la secuencia de aminoacidos en relacion con una proteina de la capside de AAV parental, que, cuando estan presentes en un virion de AAV, confieren un aumento en la infectividad de uno o mas tipos de celulas musculares en comparacion con la infectividad de las celulas musculares por un virion de AAV que comprende la proteina de la capside de AAV parental no modificada. Tambien se proporcionan viriones de AAV recombinantes y composiciones farmaceuticas de estos que comprenden una proteina de variante de capside de AAV como se describe en la presente, metodos para producir estos viriones y proteinas de la capside de rAAV, y metodos para usar estos viriones y proteinas de la capside de rAAV en la investigacion y en la practica clinica, por ejemplo, en la administracion de secuencias de acidos nucleicos a una o mas celulas musculares para el tratamiento de trastornos y enfermedades musculares
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762560901P | 2017-09-20 | 2017-09-20 | |
PCT/US2018/051812 WO2019060454A2 (en) | 2017-09-20 | 2018-09-19 | CAPSID VARIANT ADENO-ASSOCIATED VIRUSES AND METHODS OF USE |
Publications (1)
Publication Number | Publication Date |
---|---|
PE20201264A1 true PE20201264A1 (es) | 2020-11-19 |
Family
ID=65810888
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2020000407A PE20201264A1 (es) | 2017-09-20 | 2018-09-19 | Variantes de capsides de virus adenoasociados y metodos de uso de estas |
Country Status (25)
Country | Link |
---|---|
US (4) | US11118192B2 (es) |
EP (2) | EP3684423B1 (es) |
JP (1) | JP6825167B2 (es) |
KR (2) | KR20220070075A (es) |
CN (1) | CN111836649A (es) |
AU (1) | AU2018337833B2 (es) |
BR (1) | BR112020005436B1 (es) |
CA (1) | CA3076348A1 (es) |
CL (1) | CL2020000728A1 (es) |
CO (1) | CO2020004738A2 (es) |
CR (1) | CR20200165A (es) |
DK (1) | DK3684423T3 (es) |
ES (1) | ES2948902T3 (es) |
FI (1) | FI3684423T3 (es) |
IL (1) | IL273261A (es) |
MX (1) | MX2020003176A (es) |
NZ (1) | NZ763018A (es) |
PE (1) | PE20201264A1 (es) |
PH (1) | PH12020550094A1 (es) |
PT (1) | PT3684423T (es) |
RU (1) | RU2770922C2 (es) |
SG (1) | SG11202002276VA (es) |
UA (1) | UA127455C2 (es) |
WO (1) | WO2019060454A2 (es) |
ZA (1) | ZA202001679B (es) |
Families Citing this family (38)
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AU2018281280A1 (en) | 2017-06-07 | 2020-01-16 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
RU2770922C2 (ru) | 2017-09-20 | 2022-04-25 | 4Д Молекьюлар Терапьютикс Инк. | Капсиды вариантов аденоассоциированных вирусов и методы их применения |
CA3083472A1 (en) | 2017-11-27 | 2019-05-31 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
CA3111076A1 (en) | 2018-08-30 | 2020-03-05 | Tenaya Therapeutics, Inc. | Cardiac cell reprogramming with myocardin and ascl1 |
AU2019346447A1 (en) | 2018-09-26 | 2021-04-29 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
CN110437317B (zh) * | 2019-01-30 | 2023-05-02 | 上海科技大学 | 具有变异衣壳蛋白的腺相关病毒及其用途 |
WO2020198233A1 (en) * | 2019-03-25 | 2020-10-01 | The Regents Of The University Of California | Methods of treating tnni3-mediated cardiomyopathy |
JP2022527199A (ja) * | 2019-04-01 | 2022-05-31 | テナヤ セラピューティクス, インコーポレイテッド | 操作されたカプシドを有するアデノ随伴ウイルス |
US20230137971A1 (en) | 2019-07-11 | 2023-05-04 | Tenaya Therapeutics Inc. | Cardiac cell reprogramming with micrornas and other factors |
US20220389457A1 (en) * | 2019-10-23 | 2022-12-08 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
AU2020388404A1 (en) * | 2019-11-22 | 2022-06-09 | The Children's Hospital Of Philadelphia | Adeno-associated viral vector variants |
CA3171166A1 (en) | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
CN116018183A (zh) * | 2020-04-08 | 2023-04-25 | 犹他大学研究基金会 | 使用基因疗法修复心力衰竭的方法 |
CN115515613A (zh) * | 2020-04-27 | 2022-12-23 | 4D分子治疗有限公司 | 密码子优化的gla基因及其用途 |
US11900272B2 (en) | 2020-05-13 | 2024-02-13 | Factset Research System Inc. | Method and system for mapping labels in standardized tables using machine learning |
WO2021230987A1 (en) | 2020-05-13 | 2021-11-18 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
JP2023526923A (ja) * | 2020-05-14 | 2023-06-26 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ポンペ病の治療に有用な組成物 |
CN116209477A (zh) * | 2020-06-30 | 2023-06-02 | 儿童国家医疗中心 | 使用重组人酸性鞘氨醇酶改善骨骼肌纤维的修复 |
US20230313186A1 (en) * | 2020-07-24 | 2023-10-05 | The Trustees Of The University Of Pennsylvania | Trophin genome editing for treating duchenne muscular dystrophy (dmd) |
CN113754727B (zh) * | 2020-09-30 | 2022-07-12 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
US11781156B2 (en) | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
CN117062615A (zh) * | 2020-11-10 | 2023-11-14 | 阿维麦克思生物制药公司 | 工程化的病毒衣壳及使用方法 |
CN112725342B (zh) * | 2021-02-02 | 2024-05-17 | 中国科学院深圳先进技术研究院 | 一种启动子pCALM2及其应用 |
US20230062110A1 (en) * | 2021-03-26 | 2023-03-02 | Adverum Biotechnologies, Inc. | Intravitreal dosing for delivery of polynucleotides to retinal cones |
WO2022221404A2 (en) * | 2021-04-13 | 2022-10-20 | Capsida, Inc. | Adeno-associated virus compositions having increased brain enrichment |
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