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
- associated viral
- vector variants
- targeting peptides
- Prior art date
Links
Classifications
-
- 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
-
- 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
- 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)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (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)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Gastroenterology & Hepatology (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
En la presente se proveen péptidos dirigidos y vectores que contienen una secuencia que codifica los péptidos dirigidos que administran agentes a subestructuras específicas en el cerebro; en la presente se proveen vectores virales que comprenden cada uno un cápside modificado, en donde el cápside modificado comprende al menos una secuencia de aminoácidos que dirige el vector viral a una estructura cerebral distinta.
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 (12)
Country | Link |
---|---|
US (2) | US20230346981A1 (es) |
EP (1) | EP4061427A4 (es) |
JP (1) | JP2023503455A (es) |
KR (1) | KR20220116459A (es) |
CN (1) | CN115023242A (es) |
AU (1) | AU2020388404A1 (es) |
BR (1) | BR112022009864A2 (es) |
CA (1) | CA3159113A1 (es) |
CO (1) | CO2022008507A2 (es) |
IL (1) | IL293140A (es) |
MX (1) | MX2022006188A (es) |
WO (1) | WO2021102234A1 (es) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023205397A1 (en) * | 2022-04-22 | 2023-10-26 | The Children's Hospital Of Philadelphia | Human ependyma-specific promoter and uses thereof |
WO2023220287A1 (en) * | 2022-05-11 | 2023-11-16 | The Children's Hospital Of Philadelphia | Adeno-associated viral vectors for targeting deep brain structures |
WO2023220386A1 (en) * | 2022-05-13 | 2023-11-16 | The Children's Hospital Of Philadelphia | Adeno-associated viral vectors for targeting brain microvasculature |
US20230398192A1 (en) | 2022-05-16 | 2023-12-14 | Genzyme Corporation | Methods of treating metachromatic leukodystrophy |
WO2024017387A1 (en) * | 2022-07-22 | 2024-01-25 | Shanghai Vitalgen Biopharma Co., Ltd. | Novel aav capsids for targeting nervous system and uses thereof |
KR20240029316A (ko) * | 2022-08-26 | 2024-03-05 | 경희대학교 산학협력단 | 활막 표적화 화합물 및 이의 용도 |
WO2024064673A2 (en) * | 2022-09-19 | 2024-03-28 | The Children's Hospital Of Philadelphia | Aav evolution at single-cell resolution using split-seq |
Family Cites Families (8)
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 |
AU2002360291A1 (en) * | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
EP3583220A4 (en) * | 2017-02-15 | 2021-07-07 | The University of North Carolina at Chapel Hill | PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM |
US20210324354A1 (en) * | 2017-05-12 | 2021-10-21 | The Children's Hospital Of Philadelphia | Sulfamidase (sgsh) variants, vectors, compositions and methods and uses for treating mucopolysaccharidosis type iiia (mps iiia) |
CR20200165A (es) * | 2017-09-20 | 2020-06-05 | 4D Molecular Therapeutics Inc | Variantes de cápsides de virus adenoasociados y métodos de uso de estas |
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 |
US11400168B2 (en) * | 2017-12-07 | 2022-08-02 | California Institute Of Technology | Methods and systems for noninvasive control of brain cells and related vectors and compositions |
EP3793616A1 (en) * | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
-
2020
- 2020-11-20 EP EP20890214.8A patent/EP4061427A4/en active Pending
- 2020-11-20 US US17/778,783 patent/US20230346981A1/en active Pending
- 2020-11-20 KR KR1020227021143A patent/KR20220116459A/ko unknown
- 2020-11-20 MX MX2022006188A patent/MX2022006188A/es unknown
- 2020-11-20 CA CA3159113A patent/CA3159113A1/en active Pending
- 2020-11-20 BR BR112022009864A patent/BR112022009864A2/pt unknown
- 2020-11-20 AU AU2020388404A patent/AU2020388404A1/en active Pending
- 2020-11-20 CN CN202080094086.2A patent/CN115023242A/zh active Pending
- 2020-11-20 WO PCT/US2020/061464 patent/WO2021102234A1/en active Application Filing
- 2020-11-20 JP JP2022529681A patent/JP2023503455A/ja active Pending
- 2020-11-20 IL IL293140A patent/IL293140A/en unknown
-
2022
- 2022-06-16 CO CONC2022/0008507A patent/CO2022008507A2/es unknown
-
2023
- 2023-10-20 US US18/491,365 patent/US20240100194A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
EP4061427A4 (en) | 2024-01-10 |
BR112022009864A2 (pt) | 2022-08-02 |
CO2022008507A2 (es) | 2022-07-08 |
JP2023503455A (ja) | 2023-01-30 |
US20230346981A1 (en) | 2023-11-02 |
AU2020388404A1 (en) | 2022-06-09 |
WO2021102234A1 (en) | 2021-05-27 |
CN115023242A (zh) | 2022-09-06 |
EP4061427A1 (en) | 2022-09-28 |
US20240100194A1 (en) | 2024-03-28 |
KR20220116459A (ko) | 2022-08-23 |
IL293140A (en) | 2022-07-01 |
CA3159113A1 (en) | 2021-05-27 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MX2022006188A (es) | Variantes del vector viral adenoasociado. | |
MX2021006646A (es) | Vector viral adenoasociado recombinante para el suministro de genes. | |
PH12018502645A1 (en) | Optimized mini-dystrophin genes and expression cassettes and their use | |
MX2018006840A (es) | Focalizacion de peptidos para dirigir virus adenoasociados. | |
PH12018502361A1 (en) | Gdf15 fusion proteins and uses thereof | |
CL2022001372A1 (es) | Construcciones para terapia génica con microdistrofina y uso de las mismas. | |
MX2021014552A (es) | Vectores viricos y uso de los mismos en terapias celulares adoptivas. | |
EA201891137A1 (ru) | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a | |
EA201990885A1 (ru) | Композиции, содержащие короткие пептиды, полученные из pedf, и их применение | |
MX2017012097A (es) | Variantes de virus adenoasociado y métodos de uso de estas. | |
NZ737757A (en) | Peptide oligonucleotide conjugates | |
SA518390954B1 (ar) | لقاحات علاجية لفيروس الورم الحليمي البشري 18 | |
AR086998A1 (es) | Coagonistas del receptor de glucagon/glp-1 | |
NZ801728A (en) | Atf5 peptide variants and uses thereof | |
WO2014205317A3 (en) | Cyclic peptides as protein targeting agents | |
MY169703A (en) | Antimicrobial fusion compounds and uses thereof | |
MX2018015629A (es) | Sistema de vector viral adeno-asociado traslapado doble para la expresion de abc4a. | |
MX2020000676A (es) | Composiciones y metodos para tratar beta-hemoglobinopatias. | |
MX2016002937A (es) | Vacuna oncologica. | |
MX2021003420A (es) | Peptidos cationicos lipidados amino terciario para la administracion de acido nucleico. | |
MX2019006515A (es) | Composiciones que comprenden peptido wkdeagkplvk. | |
WO2017053629A3 (en) | Light-controlled gene delivery with virus vectors through incorporation of optogenetic proteins and genetic insertion of non-conformationally constrained peptides | |
MX2021007221A (es) | Factor i del complemento y cofactor del factor i del complemento, vectores que realizan codificacion para estos y uso terapeutico. | |
EA201891138A1 (ru) | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a | |
NZ734705A (en) | Herbicide resistance protein, and encoding genes and application thereof |