BR112022019304A2 - Variantes de capsídeo de aav e usos das mesmas - Google Patents
Variantes de capsídeo de aav e usos das mesmasInfo
- Publication number
- BR112022019304A2 BR112022019304A2 BR112022019304A BR112022019304A BR112022019304A2 BR 112022019304 A2 BR112022019304 A2 BR 112022019304A2 BR 112022019304 A BR112022019304 A BR 112022019304A BR 112022019304 A BR112022019304 A BR 112022019304A BR 112022019304 A2 BR112022019304 A2 BR 112022019304A2
- Authority
- BR
- Brazil
- Prior art keywords
- aav capsid
- aav
- capsid protein
- protein variants
- variants
- Prior art date
Links
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
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- 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
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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
-
- 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
Abstract
VARIANTES DE CAPSÍDEO DE AAV E USOS DAS MESMAS. A presente invenção refere-se a composições e métodos para distribuir um transgene (por exemplo, um transgene que codifica um ou mais produtos gênicos) a uma célula alvo. A descrição é baseada, em parte, em variantes da proteína do capsídeo do vírus adenoassociado (AAV) caracterizadas por tropismos para certos tipos de células (por exemplo, neurônios, células musculares, células ósseas, células cardíacas, etc.). Em algumas modalidades, os AAVs recombinantes (rAAVs) compreendendo as variantes da proteína do capsídeo (por exemplo, AAVv66, SEQ ID Nº: 1) são empacotados de forma mais eficiente do que os rAAVs com certas proteínas do capsídeo do AAV tipo selvagem. Os métodos de distribuição de um rAAV compreendendo as variantes da proteína do capsídeo de AAV também são descritos pela descrição.
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 |
---|---|
BR112022019304A2 true BR112022019304A2 (pt) | 2022-12-06 |
Family
ID=75870702
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022019304A BR112022019304A2 (pt) | 2020-03-31 | 2021-03-30 | Variantes de capsídeo de aav e usos das mesmas |
Country Status (14)
Country | Link |
---|---|
US (1) | US20230138766A1 (pt) |
EP (1) | EP4126911A1 (pt) |
JP (1) | JP2023529054A (pt) |
KR (1) | KR20230113689A (pt) |
CN (1) | CN115698039A (pt) |
AR (1) | AR122404A1 (pt) |
AU (1) | AU2021248577A1 (pt) |
BR (1) | BR112022019304A2 (pt) |
CA (1) | CA3177182A1 (pt) |
CO (1) | CO2022015313A2 (pt) |
IL (1) | IL296765A (pt) |
MX (1) | MX2022012279A (pt) |
TW (1) | TW202204377A (pt) |
WO (1) | WO2021202494A1 (pt) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
WO2024020376A1 (en) * | 2022-07-22 | 2024-01-25 | 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 |
WO1998010088A1 (en) | 1996-09-06 | 1998-03-12 | 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 |
NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
US10975391B2 (en) | 2014-04-25 | 2021-04-13 | University Of Massachusetts | Recombinant AAV vectors useful for reducing immunity against transgene products |
HUE054768T2 (hu) * | 2014-05-02 | 2021-09-28 | Genzyme Corp | AAV vektorok retina és CNS génterápiára |
US20200316221A1 (en) * | 2016-10-13 | 2020-10-08 | University Of Massachusetts | Aav capsid designs |
US11578340B2 (en) * | 2016-10-13 | 2023-02-14 | University Of Massachusetts | AAV capsid designs |
-
2021
- 2021-03-30 MX MX2022012279A patent/MX2022012279A/es unknown
- 2021-03-30 BR BR112022019304A patent/BR112022019304A2/pt unknown
- 2021-03-30 TW TW110111670A patent/TW202204377A/zh unknown
- 2021-03-30 KR KR1020227037874A patent/KR20230113689A/ko unknown
- 2021-03-30 AR ARP210100790A patent/AR122404A1/es unknown
- 2021-03-30 EP EP21724401.1A patent/EP4126911A1/en active Pending
- 2021-03-30 AU AU2021248577A patent/AU2021248577A1/en active Pending
- 2021-03-30 WO PCT/US2021/024837 patent/WO2021202494A1/en active Application Filing
- 2021-03-30 JP JP2022559694A patent/JP2023529054A/ja active Pending
- 2021-03-30 US US17/915,548 patent/US20230138766A1/en active Pending
- 2021-03-30 IL IL296765A patent/IL296765A/en unknown
- 2021-03-30 CN CN202180025786.0A patent/CN115698039A/zh active Pending
- 2021-03-30 CA CA3177182A patent/CA3177182A1/en active Pending
-
2022
- 2022-10-27 CO CONC2022/0015313A patent/CO2022015313A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
KR20230113689A (ko) | 2023-08-01 |
US20230138766A1 (en) | 2023-05-04 |
IL296765A (en) | 2022-11-01 |
TW202204377A (zh) | 2022-02-01 |
AU2021248577A1 (en) | 2022-09-29 |
AR122404A1 (es) | 2022-09-07 |
CO2022015313A2 (es) | 2023-02-16 |
CA3177182A1 (en) | 2021-10-07 |
EP4126911A1 (en) | 2023-02-08 |
JP2023529054A (ja) | 2023-07-07 |
CN115698039A (zh) | 2023-02-03 |
WO2021202494A1 (en) | 2021-10-07 |
MX2022012279A (es) | 2023-02-23 |
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