MX2022006188A - Variantes del vector viral adenoasociado. - Google Patents
Variantes del vector viral adenoasociado.Info
- Publication number
- MX2022006188A MX2022006188A MX2022006188A MX2022006188A MX2022006188A MX 2022006188 A MX2022006188 A MX 2022006188A MX 2022006188 A MX2022006188 A MX 2022006188A MX 2022006188 A MX2022006188 A MX 2022006188A MX 2022006188 A MX2022006188 A MX 2022006188A
- Authority
- MX
- Mexico
- Prior art keywords
- viral vector
- adeno
- variants
- associated viral
- targeting peptides
- 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 |
|---|---|
| MX2022006188A true MX2022006188A (es) | 2022-08-22 |
Family
ID=75980202
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2022006188A MX2022006188A (es) | 2019-11-22 | 2020-11-20 | Variantes del vector viral adenoasociado. |
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 |
|---|---|---|---|---|
| EP4136457B1 (en) * | 2020-04-17 | 2025-02-19 | Regeneron Pharmaceuticals, Inc. | Detection assays for coronavirus neutralizing antibodies |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| JP2025514794A (ja) * | 2022-04-22 | 2025-05-09 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | ヒト上衣特異的プロモーターおよびその使用方法 |
| CN119654334A (zh) * | 2022-05-11 | 2025-03-18 | 费城儿童医院 | 用于靶向深层脑结构的腺相关病毒载体 |
| AU2023267671A1 (en) * | 2022-05-13 | 2024-12-05 | The Children's Hospital Of Philadelphia | Adeno-associated viral vectors for targeting brain microvasculature |
| JP2025521111A (ja) | 2022-05-16 | 2025-07-08 | ジェンザイム・コーポレーション | 異染性白質ジストロフィーを治療する方法 |
| WO2024017387A1 (en) | 2022-07-22 | 2024-01-25 | Shanghai Vitalgen Biopharma Co., Ltd. | Novel aav capsids for targeting nervous system and uses thereof |
| KR102870168B1 (ko) * | 2022-08-26 | 2025-10-13 | 김선아 | 활막 표적화 화합물 및 이의 용도 |
| IL319666A (en) * | 2022-09-19 | 2025-05-01 | Childrens Hospital Philadelphia | AV evolution at single-cell resolution using SPLIT-SEQ |
| IL322459A (en) * | 2023-01-31 | 2025-09-01 | Childrens Hospital Philadelphia | Innovative gene therapy construct for cln2 disease |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| CN116970041B (zh) * | 2023-07-21 | 2024-02-09 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| AU2024332181A1 (en) | 2023-08-31 | 2026-02-12 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025090598A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for treatment of cln2 disease |
| WO2025090631A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for delivery of apoe2 |
| US20250152747A1 (en) * | 2023-11-15 | 2025-05-15 | Genzyme Corporation | Methods of treating gaucher disease and gba-parkinson’s disease |
| US20250152744A1 (en) * | 2023-11-15 | 2025-05-15 | Genzyme Corporation | Methods of treating neurodegenerative disorders |
| 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 |
| CN121265801A (zh) * | 2025-06-06 | 2026-01-06 | 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 |
| PT1453547T (pt) * | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| ES2739288T3 (es) * | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| 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 |
| US11905312B2 (en) * | 2017-02-15 | 2024-02-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for gene transfer across the vasculature |
| RU2019140862A (ru) * | 2017-05-12 | 2021-06-15 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia) |
| RU2770922C2 (ru) * | 2017-09-20 | 2022-04-25 | 4Д Молекьюлар Терапьютикс Инк. | Капсиды вариантов аденоассоциированных вирусов и методы их применения |
| AU2018338728B2 (en) * | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | 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 |
| WO2019222329A1 (en) * | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| WO2020014471A1 (en) * | 2018-07-11 | 2020-01-16 | The Brigham And Women's Hospital, Inc. | Methods and compositions for delivery of agents across the blood-brain barrier |
-
2020
- 2020-11-20 KR KR1020227021143A patent/KR20220116459A/ko active Pending
- 2020-11-20 BR BR112022009864A patent/BR112022009864A2/pt unknown
- 2020-11-20 MX MX2022006188A patent/MX2022006188A/es unknown
- 2020-11-20 WO PCT/US2020/061464 patent/WO2021102234A1/en not_active Ceased
- 2020-11-20 CN CN202080094086.2A patent/CN115023242B/zh active Active
- 2020-11-20 AU AU2020388404A patent/AU2020388404A1/en active Pending
- 2020-11-20 PH PH1/2022/551247A patent/PH12022551247A1/en unknown
- 2020-11-20 CN CN202510215753.2A patent/CN119978146A/zh active Pending
- 2020-11-20 JP JP2022529681A patent/JP7712271B2/ja active Active
- 2020-11-20 US US17/778,783 patent/US20230346981A1/en active Pending
- 2020-11-20 EP EP20890214.8A patent/EP4061427A4/en active Pending
- 2020-11-20 IL IL293140A patent/IL293140A/en unknown
- 2020-11-20 CA CA3159113A patent/CA3159113A1/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 |
|---|---|
| JP2025102825A (ja) | 2025-07-08 |
| CA3159113A1 (en) | 2021-05-27 |
| JP2023503455A (ja) | 2023-01-30 |
| KR20220116459A (ko) | 2022-08-23 |
| PH12022551247A1 (en) | 2023-11-13 |
| EP4061427A1 (en) | 2022-09-28 |
| WO2021102234A1 (en) | 2021-05-27 |
| BR112022009864A2 (pt) | 2022-08-02 |
| JP7712271B2 (ja) | 2025-07-23 |
| CN119978146A (zh) | 2025-05-13 |
| JP2025148448A (ja) | 2025-10-07 |
| CN115023242A (zh) | 2022-09-06 |
| CO2022008507A2 (es) | 2022-07-08 |
| EP4061427A4 (en) | 2024-01-10 |
| IL293140A (en) | 2022-07-01 |
| US20230346981A1 (en) | 2023-11-02 |
| CN115023242B (zh) | 2025-03-14 |
| US20240100194A1 (en) | 2024-03-28 |
| AU2020388404A1 (en) | 2022-06-09 |
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