MA51353B1 - Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit - Google Patents
Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduitInfo
- Publication number
- MA51353B1 MA51353B1 MA51353A MA51353A MA51353B1 MA 51353 B1 MA51353 B1 MA 51353B1 MA 51353 A MA51353 A MA 51353A MA 51353 A MA51353 A MA 51353A MA 51353 B1 MA51353 B1 MA 51353B1
- Authority
- MA
- Morocco
- Prior art keywords
- aavrh74
- aav9
- associated virus
- hybrid
- recombinant adeno
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 230000002440 hepatic effect Effects 0.000 title abstract 2
- 230000010415 tropism Effects 0.000 title abstract 2
- 108090000565 Capsid Proteins Proteins 0.000 abstract 4
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 4
- 241000649044 Adeno-associated virus 9 Species 0.000 abstract 1
- 208000026350 Inborn Genetic disease Diseases 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 208000016361 genetic disease Diseases 0.000 abstract 1
- 230000002232 neuromuscular Effects 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
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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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
- C07K14/01—DNA viruses
- C07K14/075—Adenoviridae
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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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- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Immunology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18305399 | 2018-04-05 | ||
| PCT/EP2019/058560 WO2019193119A1 (en) | 2018-04-05 | 2019-04-04 | HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MA51353A1 MA51353A1 (fr) | 2021-09-30 |
| MA51353B1 true MA51353B1 (fr) | 2022-09-30 |
Family
ID=62063468
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA51353A MA51353B1 (fr) | 2018-04-05 | 2019-04-04 | Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US12037362B2 (https=) |
| EP (2) | EP3775173A1 (https=) |
| JP (2) | JP7374119B2 (https=) |
| KR (2) | KR20210015770A (https=) |
| CN (2) | CN112004925B (https=) |
| AU (2) | AU2019249890B2 (https=) |
| BR (2) | BR112020020223A2 (https=) |
| CA (2) | CA3093347A1 (https=) |
| IL (2) | IL277740A (https=) |
| MA (1) | MA51353B1 (https=) |
| TN (1) | TN2020000187A1 (https=) |
| WO (2) | WO2019193119A1 (https=) |
| ZA (1) | ZA202005422B (https=) |
Families Citing this family (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20210363193A1 (en) * | 2018-04-27 | 2021-11-25 | Universität Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| US12466864B2 (en) | 2019-03-25 | 2025-11-11 | Genethon | Production of large-sized quasidystrophins using overlapping AAV vectors |
| CN114127088B (zh) * | 2019-04-23 | 2024-05-14 | 国家医疗保健研究所 | 变体衣壳蛋白和腺相关病毒aav变体及其药物组合物 |
| JP7651562B2 (ja) | 2019-09-19 | 2025-03-26 | ジェネトン | Ｆｋｒｐの心毒性を軽減する遺伝子治療発現系 |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| US12188039B2 (en) | 2019-11-08 | 2025-01-07 | The Board Of Regents Of The University Of Texas System | Recombinant adeno-associated viral vector for gene delivery |
| EP4073257A1 (en) * | 2019-12-09 | 2022-10-19 | UCL Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
| EP4077684A1 (en) | 2019-12-18 | 2022-10-26 | Genethon | Production of recombinant viral vectors from plant hairy roots |
| WO2021229255A1 (en) | 2020-05-14 | 2021-11-18 | Genethon | Tools and method for preventing a a¥ neutralization by antibodies |
| EP3913060A1 (en) | 2020-05-22 | 2021-11-24 | Genethon | Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy |
| CN116322790A (zh) | 2020-06-09 | 2023-06-23 | 吉尼松公司 | 用于治疗扩张型心肌病的nrf2激活剂 |
| US20230220391A1 (en) | 2020-06-09 | 2023-07-13 | Genethon | CILP-1 Inhibitors for Use in the Treatment of Dilated Cardiomyopathies |
| WO2021250076A1 (en) | 2020-06-09 | 2021-12-16 | Genethon | Treatment of genetic dilated cardiomyopathies |
| US20230256117A1 (en) | 2020-06-19 | 2023-08-17 | Genethon | Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg |
| CA3187635A1 (en) | 2020-07-03 | 2022-01-06 | Genethon | Method for engineering novel hybrid aav capsids through hypervariable regions swapping |
| EP4179096A1 (en) | 2020-07-10 | 2023-05-17 | Genethon | A novel muscle-specific promoter |
| EP4421179A3 (en) | 2020-09-10 | 2024-12-18 | Genethon | Peptide-modified aav capsid |
| US20230272433A1 (en) | 2020-09-29 | 2023-08-31 | Genethon | Enhancing Utrophin Expression in Cell by Inducing Mutations Within Utrophin Regulatory Elements and Therapeutic Use Thereof |
| KR20230117177A (ko) * | 2020-12-03 | 2023-08-07 | 유니버시티 오브 매사추세츠 | 진행성 골화성 섬유이형성증을 위한 신규 유전자 치료제의개발 |
| EP4273236A4 (en) * | 2020-12-29 | 2025-01-08 | Jiangsu Genscript Probio Biotech Co., Ltd. | HEK293T CELL STRAIN WITH HIGH DISPERSIBILITY AND SCREENING METHODS THEREFOR |
| US20240115734A1 (en) * | 2021-02-09 | 2024-04-11 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| US20240165271A1 (en) | 2021-03-26 | 2024-05-23 | The Board Of Regents Of The University Of Texas System | Nucleotide editing to reframe dmd transcripts by base editing and prime editing |
| WO2022226301A1 (en) * | 2021-04-23 | 2022-10-27 | The University Of North Carolina At Chapel Hill | Chimeric heart tropic aav capsids |
| EP4381077A1 (en) | 2021-08-04 | 2024-06-12 | Genethon | Hybrid promoters for gene expression in muscles and in the cns |
| CN115232818B (zh) * | 2021-09-18 | 2025-10-21 | 南京启真基因工程有限公司 | 构建dok7基因突变的先天性肌无力模型猪核移植供体细胞的基因编辑系统及其应用 |
| US20230211014A1 (en) * | 2021-12-29 | 2023-07-06 | Genetic Cures for Kids Inc. | AAV Gene Therapy for Spastic Paraplegia |
| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| CN119317718A (zh) | 2022-01-25 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于改善心脏转导和肝脏去靶向的aav衣壳 |
| CN114657152A (zh) * | 2022-03-31 | 2022-06-24 | 苏州博腾生物制药有限公司 | 一种新型aav血清型及其制备方法 |
| CN116925192B (zh) * | 2022-04-12 | 2025-04-29 | 合肥星眸生物科技有限公司 | 一种融合型腺相关病毒及其应用 |
| WO2023237731A1 (en) | 2022-06-09 | 2023-12-14 | Genethon | N-terminal truncated gde for the treatment of glycogen storage disease iii |
| ES3057171T3 (en) | 2022-06-10 | 2026-02-26 | Genethon | Peptide-modified aav capsid with enhanced muscle transduction efficiency |
| CN120112308A (zh) | 2022-10-07 | 2025-06-06 | 吉尼松公司 | 使用抗fap car-t细胞免疫治疗骨骼肌病 |
| EP4602055A1 (en) | 2022-10-12 | 2025-08-20 | Genethon | Hybrid aav vector enhancing transgene expression in the liver |
| CN116041443B (zh) * | 2022-12-30 | 2023-09-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2024173802A2 (en) * | 2023-02-17 | 2024-08-22 | Biogen Ma Inc. | Rgd-containing peptides for delivering payloads |
| CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| EP4677073A1 (en) | 2023-03-10 | 2026-01-14 | Genethon | Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter |
| CN121420060A (zh) | 2023-06-29 | 2026-01-27 | 吉尼松公司 | 用于治疗糖原贮积病iii的n-端截短的糖原脱支酶 |
| KR20260035966A (ko) * | 2023-07-10 | 2026-03-13 | 케이트 테라퓨틱스, 인크. | Lgmd2b/디스펄린병증을 치료하기 위한 aav 유전자 치료 접근법 |
| WO2025017168A1 (en) | 2023-07-19 | 2025-01-23 | Genethon | Novel optimized utrophin micro-genes |
| CN121605119A (zh) | 2023-07-20 | 2026-03-03 | 吉尼松公司 | 新的中型肌营养不良蛋白 |
| CN116970041B (zh) * | 2023-07-21 | 2024-02-09 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN117031014A (zh) * | 2023-08-14 | 2023-11-10 | 陕西脉元生物科技有限公司 | 抗matr3自身抗体的试剂在制备检测神经系统自身免疫疾病的产品中的应用及试剂盒 |
| WO2025072698A2 (en) * | 2023-09-27 | 2025-04-03 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Aav9 vectors and capsid polypeptides for use in treating cns disorders |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025137378A1 (en) * | 2023-12-20 | 2025-06-26 | Aavantibio, Inc. | Aav capsid engineering for cardiac and/or musculoskeletal gene therapy |
| WO2025133247A1 (en) | 2023-12-21 | 2025-06-26 | Genethon | Methods for aav vector re-administration |
| WO2025232917A1 (en) * | 2024-05-10 | 2025-11-13 | Hangzhou Jiayin Biotech Ltd. | Novel liver detargeted aav variants and uses thereof |
| WO2026017727A1 (en) | 2024-07-16 | 2026-01-22 | Genethon | Alternative barcoding method to assess transduction efficiency of aav vectors |
| WO2026041758A1 (en) | 2024-08-22 | 2026-02-26 | Genethon | Modified aav capsid with enhanced transduction efficiency |
| WO2026064736A1 (en) * | 2024-09-23 | 2026-03-26 | Carbon Biosciences, Inc. | Parvovirus compositions and related methods for gene therapy |
| WO2026064739A1 (en) * | 2024-09-23 | 2026-03-26 | Carbon Biosciences, Inc. | Parvovirus compositions and related methods for gene therapy |
| CN120842333A (zh) * | 2025-06-20 | 2025-10-28 | 上海朗昇生物科技有限公司 | 工程化的腺相关病毒衣壳蛋白和载体及其用途 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| TR201908020T4 (tr) | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| MX367100B (es) | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| NZ701693A (en) | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| EP2660325A3 (en) | 2012-05-02 | 2014-02-12 | Christian Medical College | AAV vectors and corresponding nucleotide sequences and methods |
| HK1220488A1 (zh) * | 2013-03-15 | 2017-05-05 | The Children's Hospital Of Philadelphia | 含有填充者/填充物多核苷酸序列的载体及其使用方法 |
| MX388221B (es) * | 2013-04-20 | 2025-03-19 | Res Institute At Nationwide Children´S Hospital | ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2. |
| HK1222673A1 (zh) | 2013-07-12 | 2017-07-07 | The Children's Hospital Of Philadelphia | Aav载体和用於抗aav(腺相关病毒)中和抗体的检测 |
| IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3384034B1 (en) | 2015-12-02 | 2020-07-08 | The Board of Trustees of the Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| WO2017161273A1 (en) | 2016-03-18 | 2017-09-21 | The Children's Hospital Of Philadelphia | Therapeutic for treatment of diseases including the central nervous system |
| IL305149A (en) * | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| MX2020010465A (es) * | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. |
-
2019
- 2019-04-04 MA MA51353A patent/MA51353B1/fr unknown
- 2019-04-04 BR BR112020020223-1A patent/BR112020020223A2/pt unknown
- 2019-04-04 US US16/981,086 patent/US12037362B2/en active Active
- 2019-04-04 KR KR1020207032024A patent/KR20210015770A/ko not_active Ceased
- 2019-04-04 JP JP2020554181A patent/JP7374119B2/ja active Active
- 2019-04-04 CN CN201980023352.XA patent/CN112004925B/zh active Active
- 2019-04-04 WO PCT/EP2019/058560 patent/WO2019193119A1/en not_active Ceased
- 2019-04-04 TN TNP/2020/000187A patent/TN2020000187A1/en unknown
- 2019-04-04 CA CA3093347A patent/CA3093347A1/en active Pending
- 2019-04-04 AU AU2019249890A patent/AU2019249890B2/en active Active
- 2019-04-04 EP EP19714230.0A patent/EP3775173A1/en active Pending
- 2019-10-04 JP JP2021558887A patent/JP2022526405A/ja not_active Withdrawn
- 2019-10-04 EP EP19782603.5A patent/EP3947653A1/en not_active Withdrawn
- 2019-10-04 BR BR112021019821A patent/BR112021019821A2/pt not_active Application Discontinuation
- 2019-10-04 CA CA3134183A patent/CA3134183A1/en not_active Withdrawn
- 2019-10-04 KR KR1020217031502A patent/KR20210153047A/ko not_active Withdrawn
- 2019-10-04 WO PCT/EP2019/076958 patent/WO2020200499A1/en not_active Ceased
- 2019-10-04 CN CN201980095204.9A patent/CN113795574A/zh active Pending
- 2019-10-04 AU AU2019439673A patent/AU2019439673A1/en not_active Withdrawn
- 2019-10-04 US US17/600,715 patent/US20220228167A1/en not_active Abandoned
-
2020
- 2020-08-31 ZA ZA2020/05422A patent/ZA202005422B/en unknown
- 2020-10-01 IL IL277740A patent/IL277740A/en unknown
-
2021
- 2021-10-03 IL IL286926A patent/IL286926A/en unknown
-
2024
- 2024-06-06 US US18/735,636 patent/US20240317814A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN113795574A (zh) | 2021-12-14 |
| AU2019439673A1 (en) | 2021-10-07 |
| BR112021019821A2 (pt) | 2021-12-07 |
| JP2022526405A (ja) | 2022-05-24 |
| US20220228167A1 (en) | 2022-07-21 |
| CN112004925B (zh) | 2024-09-27 |
| EP3775173A1 (en) | 2021-02-17 |
| CA3093347A1 (en) | 2019-10-10 |
| AU2019249890A1 (en) | 2020-09-24 |
| AU2019249890B2 (en) | 2024-10-10 |
| BR112020020223A2 (pt) | 2021-01-19 |
| JP2021520203A (ja) | 2021-08-19 |
| EP3947653A1 (en) | 2022-02-09 |
| IL286926A (en) | 2021-10-31 |
| WO2020200499A1 (en) | 2020-10-08 |
| US12037362B2 (en) | 2024-07-16 |
| KR20210015770A (ko) | 2021-02-10 |
| RU2020136195A (ru) | 2022-05-06 |
| MA51353A1 (fr) | 2021-09-30 |
| CA3134183A1 (en) | 2020-10-08 |
| ZA202005422B (en) | 2023-03-29 |
| JP7374119B2 (ja) | 2023-11-06 |
| KR20210153047A (ko) | 2021-12-16 |
| NZ767516A (en) | 2024-11-29 |
| US20210107948A1 (en) | 2021-04-15 |
| WO2019193119A1 (en) | 2019-10-10 |
| CN112004925A (zh) | 2020-11-27 |
| US20240317814A1 (en) | 2024-09-26 |
| TN2020000187A1 (en) | 2022-04-04 |
| IL277740A (en) | 2020-11-30 |
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