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
- 210000000234 capsid Anatomy 0.000 title abstract 2
- 108090000565 Capsid Proteins Proteins 0.000 abstract 4
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 4
- 241000702421 Dependoparvovirus Species 0.000 abstract 2
- 108700019146 Transgenes Proteins 0.000 abstract 2
- 210000004027 cell Anatomy 0.000 abstract 2
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 abstract 1
- 210000002449 bone cell Anatomy 0.000 abstract 1
- 210000002064 heart cell Anatomy 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 210000000663 muscle cell Anatomy 0.000 abstract 1
- 210000002569 neuron Anatomy 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000010415 tropism Effects 0.000 abstract 1
Classifications
-
- 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
- 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
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)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Virology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychology (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
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 (https=) |
| EP (1) | EP4126911A1 (https=) |
| JP (1) | JP2023529054A (https=) |
| KR (1) | KR20230113689A (https=) |
| CN (1) | CN115698039A (https=) |
| AR (1) | AR122404A1 (https=) |
| AU (1) | AU2021248577A1 (https=) |
| BR (1) | BR112022019304A2 (https=) |
| CA (1) | CA3177182A1 (https=) |
| CO (1) | CO2022015313A2 (https=) |
| IL (1) | IL296765A (https=) |
| MX (1) | MX2022012279A (https=) |
| TW (1) | TW202204377A (https=) |
| WO (1) | WO2021202494A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| AU2019237514A1 (en) | 2018-03-23 | 2020-10-08 | University Of Massachusetts | Gene therapeutics for treating bone disorders |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| 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 |
| AU722624B2 (en) | 1996-09-06 | 2000-08-10 | Trustees Of The University Of Pennsylvania, The | 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 |
| EP3134522B1 (en) | 2014-04-25 | 2021-10-06 | University of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| EP3526333A4 (en) * | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
| US20200316221A1 (en) * | 2016-10-13 | 2020-10-08 | University Of Massachusetts | Aav capsid designs |
-
2021
- 2021-03-30 WO PCT/US2021/024837 patent/WO2021202494A1/en not_active Ceased
- 2021-03-30 AU AU2021248577A patent/AU2021248577A1/en active Pending
- 2021-03-30 AR ARP210100790A patent/AR122404A1/es unknown
- 2021-03-30 KR KR1020227037874A patent/KR20230113689A/ko not_active Withdrawn
- 2021-03-30 CN CN202180025786.0A patent/CN115698039A/zh active Pending
- 2021-03-30 MX MX2022012279A patent/MX2022012279A/es unknown
- 2021-03-30 US US17/915,548 patent/US20230138766A1/en active Pending
- 2021-03-30 TW TW110111670A patent/TW202204377A/zh unknown
- 2021-03-30 CA CA3177182A patent/CA3177182A1/en active Pending
- 2021-03-30 JP JP2022559694A patent/JP2023529054A/ja active Pending
- 2021-03-30 IL IL296765A patent/IL296765A/en unknown
- 2021-03-30 BR BR112022019304A patent/BR112022019304A2/pt unknown
- 2021-03-30 EP EP21724401.1A patent/EP4126911A1/en active Pending
-
2022
- 2022-10-27 CO CONC2022/0015313A patent/CO2022015313A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4126911A1 (en) | 2023-02-08 |
| KR20230113689A (ko) | 2023-08-01 |
| CA3177182A1 (en) | 2021-10-07 |
| US20230138766A1 (en) | 2023-05-04 |
| IL296765A (en) | 2022-11-01 |
| TW202204377A (zh) | 2022-02-01 |
| CO2022015313A2 (es) | 2023-02-16 |
| WO2021202494A1 (en) | 2021-10-07 |
| MX2022012279A (es) | 2023-02-23 |
| AR122404A1 (es) | 2022-09-07 |
| JP2023529054A (ja) | 2023-07-07 |
| CN115698039A (zh) | 2023-02-03 |
| AU2021248577A1 (en) | 2022-09-29 |
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