BR112022009864A2 - Variantes do vetor viral adenoassociado - Google Patents
Variantes do vetor viral adenoassociadoInfo
- Publication number
- BR112022009864A2 BR112022009864A2 BR112022009864A BR112022009864A BR112022009864A2 BR 112022009864 A2 BR112022009864 A2 BR 112022009864A2 BR 112022009864 A BR112022009864 A BR 112022009864A BR 112022009864 A BR112022009864 A BR 112022009864A BR 112022009864 A2 BR112022009864 A2 BR 112022009864A2
- Authority
- BR
- Brazil
- Prior art keywords
- viral vector
- variants
- associated viral
- targeting peptides
- modified capsid
- Prior art date
Links
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
- 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/04—Linear peptides containing only normal peptide links
- C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
-
- 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
- C07K2319/00—Fusion polypeptide
- C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
-
- 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)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962939315P | 2019-11-22 | 2019-11-22 | |
| US202063084709P | 2020-09-29 | 2020-09-29 | |
| PCT/US2020/061464 WO2021102234A1 (en) | 2019-11-22 | 2020-11-20 | Adeno-associated viral vector variants |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022009864A2 true BR112022009864A2 (pt) | 2022-08-02 |
Family
ID=75980202
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022009864A BR112022009864A2 (pt) | 2019-11-22 | 2020-11-20 | Variantes do vetor viral adenoassociado |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20230346981A1 (https=) |
| EP (1) | EP4061427A4 (https=) |
| JP (3) | JP7712271B2 (https=) |
| KR (1) | KR20220116459A (https=) |
| CN (2) | CN115023242B (https=) |
| AU (1) | AU2020388404A1 (https=) |
| BR (1) | BR112022009864A2 (https=) |
| CA (1) | CA3159113A1 (https=) |
| CO (1) | CO2022008507A2 (https=) |
| IL (1) | IL293140A (https=) |
| MX (1) | MX2022006188A (https=) |
| PH (1) | PH12022551247A1 (https=) |
| WO (1) | WO2021102234A1 (https=) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2022013030A (es) * | 2020-04-17 | 2023-03-09 | Regeneron Pharma | Ensayos de deteccion para anticuerpos neutralizantes de coronavirus. |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| WO2023205397A1 (en) * | 2022-04-22 | 2023-10-26 | The Children's Hospital Of Philadelphia | Human ependyma-specific promoter and uses thereof |
| AU2023269646A1 (en) * | 2022-05-11 | 2024-12-05 | The Children's Hospital Of Philadelphia | Adeno-associated viral vectors for targeting deep brain structures |
| CN119562959A (zh) * | 2022-05-13 | 2025-03-04 | 费城儿童医院 | 用于靶向脑微脉管系统的腺相关病毒载体 |
| AU2023273809A1 (en) | 2022-05-16 | 2025-01-02 | Genzyme Corporation | Methods of treating metachromatic leukodystrophy |
| CN119894920A (zh) | 2022-07-22 | 2025-04-25 | 上海天泽云泰生物医药有限公司 | 用于靶向神经系统的新型aav衣壳及其用途 |
| KR102870168B1 (ko) * | 2022-08-26 | 2025-10-13 | 김선아 | 활막 표적화 화합물 및 이의 용도 |
| JP2025532627A (ja) * | 2022-09-19 | 2025-10-01 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | ＳＰＬｉＴ－ｓｅｑを用いたシングルセル解像でのＡＡＶ進化 |
| IL322459A (en) * | 2023-01-31 | 2025-09-01 | Childrens Hospital Philadelphia | Innovative gene therapy construct for cln2 disease |
| TW202502803A (zh) | 2023-03-10 | 2025-01-16 | 美商戴諾治療公司 | 衣殼多肽及其使用方法 |
| CN118005749B (zh) * | 2023-07-21 | 2024-07-16 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| TW202525832A (zh) | 2023-08-31 | 2025-07-01 | 美商戴諾治療公司 | 衣殼多肽及其使用方法(二) |
| WO2025090631A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for delivery of apoe2 |
| WO2025090598A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for treatment of cln2 disease |
| WO2025106874A1 (en) * | 2023-11-15 | 2025-05-22 | Genzyme Corporation | Methods of treating neurodegenerative disorders |
| US20250152747A1 (en) | 2023-11-15 | 2025-05-15 | Genzyme Corporation | Methods of treating gaucher disease and gba-parkinson’s disease |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2025213097A1 (en) * | 2024-04-05 | 2025-10-09 | The Children's Hospital Of Philadelphia | Novel gene therapy constructs for stxbp1 haploinsufficiency |
| WO2025231406A1 (en) * | 2024-05-02 | 2025-11-06 | The Children's Hospital Of Philadelphia | Methods to increase transduction of ependyma cells in brain |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| CN120242044B (zh) * | 2025-06-06 | 2025-09-23 | 昆明理工大学 | 一种靶向性的药物递送载体及其制备方法 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040031072A1 (en) * | 1999-05-06 | 2004-02-12 | La Rosa Thomas J. | Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement |
| DK2359869T3 (en) * | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US9585971B2 (en) * | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| KR102423442B1 (ko) * | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| BR112019016769A2 (pt) * | 2017-02-15 | 2020-05-26 | The University Of North Carolina At Chapel Hill | Métodos e composições para transferência genética através da vasculatura |
| MX2019013528A (es) * | 2017-05-12 | 2020-07-14 | The Children´S Hospital Of Philadelphia | Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia). |
| SG11202002276VA (en) * | 2017-09-20 | 2020-04-29 | 4D Molecular Therapeutics Inc | Adeno-associated virus variant capsids and methods of use thereof |
| US20200263199A1 (en) * | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| WO2019113538A2 (en) * | 2017-12-07 | 2019-06-13 | California Institute Of Technology | Methods and systems for noninvasive control of brain cells and related vectors and compositions |
| TW202015742A (zh) * | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒（ａａｖ）之組成物和方法 |
| CN112703198B (zh) * | 2018-07-11 | 2025-05-30 | 布里格姆妇女医院 | 用于跨血脑屏障递送试剂的方法和组合物 |
-
2020
- 2020-11-20 PH PH1/2022/551247A patent/PH12022551247A1/en unknown
- 2020-11-20 JP JP2022529681A patent/JP7712271B2/ja active Active
- 2020-11-20 IL IL293140A patent/IL293140A/en unknown
- 2020-11-20 CN CN202080094086.2A patent/CN115023242B/zh active Active
- 2020-11-20 US US17/778,783 patent/US20230346981A1/en active Pending
- 2020-11-20 KR KR1020227021143A patent/KR20220116459A/ko active Pending
- 2020-11-20 CN CN202510215753.2A patent/CN119978146A/zh active Pending
- 2020-11-20 AU AU2020388404A patent/AU2020388404A1/en active Pending
- 2020-11-20 WO PCT/US2020/061464 patent/WO2021102234A1/en not_active Ceased
- 2020-11-20 BR BR112022009864A patent/BR112022009864A2/pt unknown
- 2020-11-20 CA CA3159113A patent/CA3159113A1/en active Pending
- 2020-11-20 MX MX2022006188A patent/MX2022006188A/es unknown
- 2020-11-20 EP EP20890214.8A patent/EP4061427A4/en active Pending
-
2022
- 2022-06-16 CO CONC2022/0008507A patent/CO2022008507A2/es unknown
-
2023
- 2023-10-20 US US18/491,365 patent/US20240100194A1/en active Pending
-
2025
- 2025-03-19 JP JP2025044678A patent/JP2025102825A/ja active Pending
- 2025-07-10 JP JP2025116303A patent/JP2025148448A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023503455A (ja) | 2023-01-30 |
| US20230346981A1 (en) | 2023-11-02 |
| EP4061427A4 (en) | 2024-01-10 |
| JP2025148448A (ja) | 2025-10-07 |
| WO2021102234A1 (en) | 2021-05-27 |
| JP2025102825A (ja) | 2025-07-08 |
| CN115023242A (zh) | 2022-09-06 |
| MX2022006188A (es) | 2022-08-22 |
| JP7712271B2 (ja) | 2025-07-23 |
| CN119978146A (zh) | 2025-05-13 |
| PH12022551247A1 (en) | 2023-11-13 |
| CO2022008507A2 (es) | 2022-07-08 |
| CA3159113A1 (en) | 2021-05-27 |
| CN115023242B (zh) | 2025-03-14 |
| US20240100194A1 (en) | 2024-03-28 |
| IL293140A (en) | 2022-07-01 |
| AU2020388404A1 (en) | 2022-06-09 |
| KR20220116459A (ko) | 2022-08-23 |
| EP4061427A1 (en) | 2022-09-28 |
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