MX2022012279A - Variantes de capsides de aav y usos de los mismos. - Google Patents
Variantes de capsides de aav y usos de los mismos.Info
- Publication number
- MX2022012279A MX2022012279A MX2022012279A MX2022012279A MX2022012279A MX 2022012279 A MX2022012279 A MX 2022012279A MX 2022012279 A MX2022012279 A MX 2022012279A MX 2022012279 A MX2022012279 A MX 2022012279A MX 2022012279 A MX2022012279 A MX 2022012279A
- Authority
- MX
- Mexico
- Prior art keywords
- protein variants
- capsid protein
- disclosure
- aav
- variants
- Prior art date
Links
Classifications
-
- 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
- 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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
-
- 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)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Abstract
Aspectos de la divulgación se refieren a composiciones y métodos para entrega de un transgén (por ejemplo, un transgén que codifica uno o más productos génicos) a una célula blanco. La divulgación se basa, en parte, en variantes de la proteína de la cápside del virus adenoasociado (AAV) caracterizadas por tropismos para ciertos tipos de células (por ejemplo, neuronas, células musculares, células óseas, células cardíacas, etc.). En algunas modalidades, los AAV recombinantes (rAAV) que comprenden las variantes de la proteína de la cápside (por ejemplo, AAVv66, SEQ ID NO: 1) se empacan de manera más eficiente que los rAAV que tienen ciertas proteínas de la cápside de AAV tipo silvestre. Los métodos para entrega de un rAAV que comprende las variantes de proteína de la cápside de AAV también se describen en la divulgación.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063003143P | 2020-03-31 | 2020-03-31 | |
| PCT/US2021/024837 WO2021202494A1 (en) | 2020-03-31 | 2021-03-30 | Aav capsids variants and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2022012279A true MX2022012279A (es) | 2023-02-23 |
Family
ID=75870702
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2022012279A MX2022012279A (es) | 2020-03-31 | 2021-03-30 | Variantes de capsides de aav y usos de los mismos. |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20230138766A1 (es) |
| EP (1) | EP4126911A1 (es) |
| JP (2) | JP2023529054A (es) |
| KR (1) | KR20230113689A (es) |
| CN (1) | CN115698039A (es) |
| AR (1) | AR122404A1 (es) |
| AU (1) | AU2021248577A1 (es) |
| BR (1) | BR112022019304A2 (es) |
| CA (1) | CA3177182A1 (es) |
| CO (1) | CO2022015313A2 (es) |
| IL (1) | IL296765A (es) |
| MX (1) | MX2022012279A (es) |
| TW (2) | TW202204377A (es) |
| WO (1) | WO2021202494A1 (es) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| EP4169576A1 (en) | 2018-03-23 | 2023-04-26 | University of Massachusetts | Gene therapeutics for treating bone disorders |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| US20260028624A1 (en) * | 2022-07-22 | 2026-01-29 | University Of Massachusetts | Aav-mediated delivery of osteoblast/osteoclast-regulating mirnas for osteoporosis therapy |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5549910A (en) | 1989-03-31 | 1996-08-27 | The Regents Of The University Of California | Preparation of liposome and lipid complex compositions |
| US5252334A (en) | 1989-09-08 | 1993-10-12 | Cygnus Therapeutic Systems | Solid matrix system for transdermal drug delivery |
| JP3218637B2 (ja) | 1990-07-26 | 2001-10-15 | 大正製薬株式会社 | 安定なリポソーム水懸濁液 |
| JP2958076B2 (ja) | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| US5741516A (en) | 1994-06-20 | 1998-04-21 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| US5697899A (en) | 1995-02-07 | 1997-12-16 | Gensia | Feedback controlled drug delivery system |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| US5738868A (en) | 1995-07-18 | 1998-04-14 | Lipogenics Ltd. | Liposome compositions and kits therefor |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| US5656016A (en) | 1996-03-18 | 1997-08-12 | Abbott Laboratories | Sonophoretic drug delivery system |
| US5797898A (en) | 1996-07-02 | 1998-08-25 | Massachusetts Institute Of Technology | Microchip drug delivery devices |
| US5783208A (en) | 1996-07-19 | 1998-07-21 | Theratech, Inc. | Transdermal drug delivery matrix for coadministering estradiol and another steroid |
| US5779708A (en) | 1996-08-15 | 1998-07-14 | Cyberdent, Inc. | Intraosseous drug delivery device and method |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6177403B1 (en) | 1996-10-21 | 2001-01-23 | The Trustees Of The University Of Pennsylvania | Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| LT3137497T (lt) * | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| KR20190075964A (ko) * | 2016-10-13 | 2019-07-01 | 유니버시티 오브 매사추세츠 | Aav 캡시드 설계 |
| US20200316221A1 (en) * | 2016-10-13 | 2020-10-08 | University Of Massachusetts | Aav capsid designs |
-
2021
- 2021-03-30 BR BR112022019304A patent/BR112022019304A2/pt unknown
- 2021-03-30 AU AU2021248577A patent/AU2021248577A1/en active Pending
- 2021-03-30 CA CA3177182A patent/CA3177182A1/en active Pending
- 2021-03-30 US US17/915,548 patent/US20230138766A1/en active Pending
- 2021-03-30 WO PCT/US2021/024837 patent/WO2021202494A1/en not_active Ceased
- 2021-03-30 IL IL296765A patent/IL296765A/en unknown
- 2021-03-30 TW TW110111670A patent/TW202204377A/zh unknown
- 2021-03-30 EP EP21724401.1A patent/EP4126911A1/en active Pending
- 2021-03-30 MX MX2022012279A patent/MX2022012279A/es unknown
- 2021-03-30 KR KR1020227037874A patent/KR20230113689A/ko not_active Withdrawn
- 2021-03-30 AR ARP210100790A patent/AR122404A1/es unknown
- 2021-03-30 CN CN202180025786.0A patent/CN115698039A/zh active Pending
- 2021-03-30 TW TW114144710A patent/TW202609061A/zh unknown
- 2021-03-30 JP JP2022559694A patent/JP2023529054A/ja not_active Withdrawn
-
2022
- 2022-10-27 CO CONC2022/0015313A patent/CO2022015313A2/es unknown
-
2026
- 2026-04-02 JP JP2026061258A patent/JP2026108839A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4126911A1 (en) | 2023-02-08 |
| TW202609061A (zh) | 2026-03-01 |
| JP2023529054A (ja) | 2023-07-07 |
| BR112022019304A2 (pt) | 2022-12-06 |
| IL296765A (en) | 2022-11-01 |
| CN115698039A (zh) | 2023-02-03 |
| US20230138766A1 (en) | 2023-05-04 |
| TW202204377A (zh) | 2022-02-01 |
| AR122404A1 (es) | 2022-09-07 |
| AU2021248577A1 (en) | 2022-09-29 |
| JP2026108839A (ja) | 2026-06-30 |
| CO2022015313A2 (es) | 2023-02-16 |
| KR20230113689A (ko) | 2023-08-01 |
| CA3177182A1 (en) | 2021-10-07 |
| WO2021202494A1 (en) | 2021-10-07 |
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