CR20200165A - Variantes de cápsides de virus adenoasociados y métodos de uso de estas - Google Patents
Variantes de cápsides de virus adenoasociados y métodos de uso de estasInfo
- Publication number
- CR20200165A CR20200165A CR20200165A CR20200165A CR20200165A CR 20200165 A CR20200165 A CR 20200165A CR 20200165 A CR20200165 A CR 20200165A CR 20200165 A CR20200165 A CR 20200165A CR 20200165 A CR20200165 A CR 20200165A
- Authority
- CR
- Costa Rica
- Prior art keywords
- aav
- methods
- virions
- muscle cells
- associated virus
- Prior art date
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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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- 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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- 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
- A61K38/44—Oxidoreductases (1)
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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
- 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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- C07—ORGANIC CHEMISTRY
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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
- C12N15/86—Viral vectors
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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
- C12N15/861—Adenoviral vectors
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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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- 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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- 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
- 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
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- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- 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
- C12N2750/14121—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
- 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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- 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
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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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- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
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Abstract
En la presente se proporcionan proteínas de cápsides variantes de virus adenoasociados (AAV) que tienen una o más modificaciones en la secuencia de aminoácidos en relación con una proteína de la cápside de AAV parental, que, cuando están presentes en un virión de AAV, confieren un aumento en la infectividad de uno o más tipos de células musculares en comparación con la infectividad de las células musculares por un virión de AAV que comprende la proteína de la cápside de AAV parental no modificada. También se proporcionan viriones de AAV recombinantes y composiciones farmacéuticas de estos que comprenden una proteína de variante de cápside de AAV como se describe en la presente, métodos para producir estos viriones y proteínas de la cápside de rAAV, y métodos para usar estos viriones y proteínas de cápside de rAAV en la investigación y en la práctica clínica, por ejemplo, en la administración de secuencias de ácidos nucleicos a una o más células 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 |
---|---|
CR20200165A true CR20200165A (es) | 2020-06-05 |
Family
ID=65810888
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CR20200165A CR20200165A (es) | 2017-09-20 | 2018-09-19 | Variantes de cápsides de virus adenoasociados y métodos de uso de estas |
Country Status (25)
Country | Link |
---|---|
US (4) | US11118192B2 (es) |
EP (2) | EP4218828A3 (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 (41)
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WO2018226861A1 (en) | 2017-06-07 | 2018-12-13 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
KR20220070075A (ko) * | 2017-09-20 | 2022-05-27 | 4디 몰레큘러 테라퓨틱스 아이엔씨. | 아데노-관련 바이러스 변이체 캡시드 및 그 용도 |
CN111770999A (zh) | 2017-11-27 | 2020-10-13 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和用于抑制血管生成的应用 |
KR20210102870A (ko) | 2018-08-30 | 2021-08-20 | 테나야 테라퓨틱스, 인코포레이티드 | 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍 |
WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | 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 |
WO2020205889A1 (en) * | 2019-04-01 | 2020-10-08 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
EP3997226A1 (en) | 2019-07-11 | 2022-05-18 | 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 |
EP4103214A2 (en) | 2020-02-13 | 2022-12-21 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
US20230108316A1 (en) * | 2020-04-08 | 2023-04-06 | University Of Utah Research Foundation | Methods for rehabilitating heart failure using gene therapy |
WO2021222094A1 (en) * | 2020-04-27 | 2021-11-04 | 4D Molecular Therapeutics Inc. | Codon optimized gla genes and uses thereof |
US11900272B2 (en) | 2020-05-13 | 2024-02-13 | Factset Research System Inc. | Method and system for mapping labels in standardized tables using machine learning |
BR112022023106A2 (pt) | 2020-05-13 | 2023-01-17 | Voyager Therapeutics Inc | Redirecionamento de tropismo de capsídeos de aav |
EP4149519A4 (en) * | 2020-05-14 | 2024-06-12 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE |
JP2023532923A (ja) * | 2020-06-30 | 2023-08-01 | チルドレンズ ナショナル メディカル センター | 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用 |
WO2022020806A2 (en) * | 2020-07-24 | 2022-01-27 | The Trustees Of The University Of Pennsylvania | Utrophin genome editing for treating duchenne muscular dystrophy (dmd) |
CN113754728B (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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