ZA202105975B - Variant aav capsids for intravitreal delivery - Google Patents
Variant aav capsids for intravitreal deliveryInfo
- Publication number
- ZA202105975B ZA202105975B ZA2021/05975A ZA202105975A ZA202105975B ZA 202105975 B ZA202105975 B ZA 202105975B ZA 2021/05975 A ZA2021/05975 A ZA 2021/05975A ZA 202105975 A ZA202105975 A ZA 202105975A ZA 202105975 B ZA202105975 B ZA 202105975B
- Authority
- ZA
- South Africa
- Prior art keywords
- variant aav
- aav capsids
- intravitreal delivery
- variant
- aav
- Prior art date
Links
- 210000000234 capsid Anatomy 0.000 title 1
- 108090000565 Capsid Proteins Proteins 0.000 abstract 2
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 210000002845 virion Anatomy 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 238000011321 prophylaxis Methods 0.000 abstract 1
- 238000011282 treatment Methods 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Virology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Ophthalmology & Optometry (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962839548P | 2019-04-26 | 2019-04-26 | |
| US201962923924P | 2019-10-21 | 2019-10-21 | |
| PCT/US2020/029895 WO2020219933A1 (en) | 2019-04-26 | 2020-04-24 | Variant aav capsids for intravitreal delivery |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ZA202105975B true ZA202105975B (en) | 2024-12-18 |
Family
ID=70740759
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ZA2021/05975A ZA202105975B (en) | 2019-04-26 | 2021-08-19 | Variant aav capsids for intravitreal delivery |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US11510950B2 (https=) |
| EP (1) | EP3958911A1 (https=) |
| JP (1) | JP7623703B2 (https=) |
| KR (1) | KR20220004114A (https=) |
| CN (2) | CN113766934B (https=) |
| AU (1) | AU2020262948A1 (https=) |
| BR (1) | BR112021020957A2 (https=) |
| CA (1) | CA3130217A1 (https=) |
| IL (1) | IL287438A (https=) |
| MX (2) | MX2021012311A (https=) |
| SG (1) | SG11202108928XA (https=) |
| TW (2) | TW202532428A (https=) |
| WO (1) | WO2020219933A1 (https=) |
| ZA (1) | ZA202105975B (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020041498A1 (en) * | 2018-08-21 | 2020-02-27 | Massachusetts Eye And Ear Infirmary | Compositions and methods for modulating transduction efficiency of adeno-associated viruses |
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| TWI900553B (zh) | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
| CN115044614B (zh) * | 2021-03-09 | 2023-10-20 | 上海目镜生物医药科技有限公司 | 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用 |
| US12385064B2 (en) * | 2021-03-26 | 2025-08-12 | Adverum Biotechnologies, Inc. | Intravitreal dosing for delivery of polynucleotides to retinal cones |
| CN113648432B (zh) * | 2021-03-31 | 2024-01-19 | 中山大学中山眼科中心 | rAAV2/Retro作为针对视网膜感光细胞的递送系统及其在制备治疗视网膜疾病药物中的应用 |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| EP4688807A1 (en) * | 2023-03-31 | 2026-02-11 | Exegenesis Bio Singapore Pte. Ltd. | Variant aav9 capsid proteins and uses thereof |
| EP4689184A1 (en) | 2023-04-05 | 2026-02-11 | Adverum Biotechnologies, Inc. | Immune landscape signatures for ocular inflammation |
| KR20260039752A (ko) | 2023-07-13 | 2026-03-20 | 크리야 테라퓨틱스, 인크. | 보체 경로 억제제 융합체 및 그의 사용 방법 |
| CN117247434B (zh) * | 2023-11-10 | 2024-02-02 | 上海朗昇生物科技有限公司 | 衣壳修饰型病毒载体及其制备和用途 |
| KR20260014488A (ko) * | 2024-07-22 | 2026-01-30 | 한국생명공학연구원 | 변이체 캡시드를 포함하는 아데노-연관 바이러스 및 이의 용도 |
| CN118955654B (zh) * | 2024-10-15 | 2025-05-16 | 朗信启昇(苏州)生物制药有限公司 | 具有变异衣壳的腺相关病毒病毒体及其用途 |
| WO2026084101A1 (ko) * | 2024-10-18 | 2026-04-23 | 주식회사 아바타테라퓨틱스 | 아데노연관바이러스 변이체 |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| WO1994004679A1 (en) | 1991-06-14 | 1994-03-03 | Genentech, Inc. | Method for making humanized antibodies |
| CA2158745C (en) | 1993-03-25 | 2007-06-19 | Richard L. Kendall | Inhibitor of vascular endothelial cell growth factor |
| KR100816621B1 (ko) | 1997-04-07 | 2008-03-24 | 제넨테크, 인크. | 항-ｖｅｇｆ 항체 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US7087411B2 (en) | 1999-06-08 | 2006-08-08 | Regeneron Pharmaceuticals, Inc. | Fusion protein capable of binding VEGF |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| US7510872B2 (en) | 2003-02-26 | 2009-03-31 | Nationwide Children's Hospital | Recombinant adeno-associated virus production |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| EP1919497B1 (en) | 2005-07-22 | 2020-02-12 | The Board of Trustees of the Leland Stanford Junior University | Light-activated cation channel and uses thereof |
| CN101484005A (zh) | 2006-05-04 | 2009-07-15 | 韦恩州立大学 | 通过向体内递送视紫红质核酸恢复视觉响应 |
| EP2287323A1 (en) * | 2009-07-31 | 2011-02-23 | Association Institut de Myologie | Widespread gene delivery to the retina using systemic administration of AAV vectors |
| US8586526B2 (en) | 2010-05-17 | 2013-11-19 | Sangamo Biosciences, Inc. | DNA-binding proteins and uses thereof |
| CN105755044A (zh) * | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| TWI698240B (zh) | 2012-05-15 | 2020-07-11 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| TWI806150B (zh) | 2014-11-07 | 2023-06-21 | 瑞士商諾華公司 | 穩定的含有高濃度抗vegf抗體之蛋白質溶液調配物 |
| US11021519B2 (en) * | 2015-03-02 | 2021-06-01 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| AU2017259968A1 (en) | 2016-05-03 | 2018-11-15 | Adverum Biotechnologies, Inc. | Organotypic pig retina culture system |
| US11364308B2 (en) * | 2016-05-13 | 2022-06-21 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| AU2017302013B2 (en) | 2016-07-29 | 2022-05-26 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CA3040179A1 (en) * | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| JP2020510424A (ja) * | 2017-02-28 | 2020-04-09 | アドヴェラム バイオテクノロジーズ， インコーポレイテッド | 改変ａａｖカプシドおよびその使用 |
-
2020
- 2020-04-23 TW TW113141337A patent/TW202532428A/zh unknown
- 2020-04-23 TW TW109113631A patent/TWI862583B/zh active
- 2020-04-24 WO PCT/US2020/029895 patent/WO2020219933A1/en not_active Ceased
- 2020-04-24 KR KR1020217038123A patent/KR20220004114A/ko active Pending
- 2020-04-24 CN CN202080031274.0A patent/CN113766934B/zh active Active
- 2020-04-24 CN CN202411569264.9A patent/CN119708162A/zh active Pending
- 2020-04-24 US US16/858,042 patent/US11510950B2/en active Active
- 2020-04-24 CA CA3130217A patent/CA3130217A1/en active Pending
- 2020-04-24 AU AU2020262948A patent/AU2020262948A1/en active Pending
- 2020-04-24 BR BR112021020957A patent/BR112021020957A2/pt unknown
- 2020-04-24 JP JP2021561683A patent/JP7623703B2/ja active Active
- 2020-04-24 SG SG11202108928XA patent/SG11202108928XA/en unknown
- 2020-04-24 MX MX2021012311A patent/MX2021012311A/es unknown
- 2020-04-24 EP EP20726600.8A patent/EP3958911A1/en active Pending
-
2021
- 2021-08-19 ZA ZA2021/05975A patent/ZA202105975B/en unknown
- 2021-10-07 MX MX2025015023A patent/MX2025015023A/es unknown
- 2021-10-20 IL IL287438A patent/IL287438A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3958911A1 (en) | 2022-03-02 |
| TWI862583B (zh) | 2024-11-21 |
| TW202532428A (zh) | 2025-08-16 |
| MX2021012311A (es) | 2021-11-12 |
| CA3130217A1 (en) | 2020-10-29 |
| CN119708162A (zh) | 2025-03-28 |
| KR20220004114A (ko) | 2022-01-11 |
| MX2025015023A (es) | 2026-02-03 |
| WO2020219933A1 (en) | 2020-10-29 |
| IL287438A (en) | 2021-12-01 |
| TW202106699A (zh) | 2021-02-16 |
| JP2022529775A (ja) | 2022-06-24 |
| US20200338146A1 (en) | 2020-10-29 |
| CN113766934A (zh) | 2021-12-07 |
| SG11202108928XA (en) | 2021-09-29 |
| US11510950B2 (en) | 2022-11-29 |
| CN113766934B (zh) | 2024-11-08 |
| BR112021020957A2 (pt) | 2021-12-14 |
| AU2020262948A1 (en) | 2021-09-30 |
| JP7623703B2 (ja) | 2025-01-29 |
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