CL2021003295A1 - Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas. - Google Patents
Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas.Info
- Publication number
- CL2021003295A1 CL2021003295A1 CL2021003295A CL2021003295A CL2021003295A1 CL 2021003295 A1 CL2021003295 A1 CL 2021003295A1 CL 2021003295 A CL2021003295 A CL 2021003295A CL 2021003295 A CL2021003295 A CL 2021003295A CL 2021003295 A1 CL2021003295 A1 CL 2021003295A1
- Authority
- CL
- Chile
- Prior art keywords
- adeno
- methods
- associated virus
- gene transfer
- virus compositions
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 4
- 101150007653 Arsa gene Proteins 0.000 title abstract 2
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 102100022146 Arylsulfatase A Human genes 0.000 abstract 2
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 abstract 2
- 238000004806 packaging method and process Methods 0.000 abstract 1
- 229920001184 polypeptide Polymers 0.000 abstract 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0276—Knock-out vertebrates
-
- 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
-
- 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
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
-
- 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
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06001—Arylsulfatase (3.1.6.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Neurosurgery (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Psychiatry (AREA)
- Environmental Sciences (AREA)
- Animal Husbandry (AREA)
- Biodiversity & Conservation Biology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
En la presente descripción se proporcionan composiciones de virus adenoasociados (AAV) que pueden expresar un polipéptido de arilsulfatasa A (ARSA) en una célula, para restablecer de ese modo la función del gen de ARSA. También se proporcionan métodos para usar las composiciones de AAV y sistemas de empaquetamiento para preparar las composiciones de AAV.
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962859539P | 2019-06-10 | 2019-06-10 | |
US201962866374P | 2019-06-25 | 2019-06-25 | |
US201962915523P | 2019-10-15 | 2019-10-15 | |
US202062960487P | 2020-01-13 | 2020-01-13 | |
US202062987858P | 2020-03-10 | 2020-03-10 | |
US202063010970P | 2020-04-16 | 2020-04-16 |
Publications (1)
Publication Number | Publication Date |
---|---|
CL2021003295A1 true CL2021003295A1 (es) | 2022-09-23 |
Family
ID=73782242
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2021003295A CL2021003295A1 (es) | 2019-06-10 | 2021-12-10 | Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas. |
Country Status (15)
Country | Link |
---|---|
US (1) | US20220204991A1 (es) |
EP (1) | EP3980447A4 (es) |
JP (1) | JP2022536338A (es) |
KR (1) | KR20220035107A (es) |
CN (1) | CN114502575A (es) |
AU (1) | AU2020292256B2 (es) |
BR (1) | BR112021024855A2 (es) |
CA (1) | CA3142932A1 (es) |
CL (1) | CL2021003295A1 (es) |
CO (1) | CO2021016797A2 (es) |
IL (1) | IL288863A (es) |
MX (1) | MX2021015076A (es) |
PE (1) | PE20220233A1 (es) |
TW (1) | TW202112807A (es) |
WO (1) | WO2020251954A1 (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023225481A1 (en) * | 2022-05-16 | 2023-11-23 | Genzyme Corporation | Methods of treating metachromatic leukodystrophy |
WO2024026494A1 (en) | 2022-07-29 | 2024-02-01 | Regeneron Pharmaceuticals, Inc. | Viral particles retargeted to transferrin receptor 1 |
WO2024036250A2 (en) * | 2022-08-10 | 2024-02-15 | Homology Medicines, Inc. | Adeno-associated virus compositions for arsa gene transfer and methods of use thereof |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9839696B2 (en) * | 2010-04-30 | 2017-12-12 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
CN107828820B (zh) * | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
MX2016014220A (es) * | 2014-05-02 | 2017-02-06 | Genzyme Corp | Vectores de aav para la terapia genica de la retina y el snc. |
EP3632923A1 (en) * | 2015-01-16 | 2020-04-08 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
WO2016154344A1 (en) * | 2015-03-24 | 2016-09-29 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
HUE057795T2 (hu) * | 2015-05-15 | 2022-06-28 | Regenxbio Inc | Adenoasszociált vírus terápiás szerek központi idegrendszerbe történõ bejuttatására |
AU2019215150A1 (en) * | 2018-02-01 | 2020-09-24 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
CA3129321A1 (en) * | 2019-02-15 | 2020-08-20 | Generation Bio Co. | Modulation of rep protein activity in closed-ended dna (cedna) production |
CN114072129A (zh) * | 2019-05-03 | 2022-02-18 | 宾夕法尼亚州大学信托人 | 可用于治疗异染性脑白质营养不良的组合物 |
-
2020
- 2020-06-09 MX MX2021015076A patent/MX2021015076A/es unknown
- 2020-06-09 KR KR1020227000707A patent/KR20220035107A/ko unknown
- 2020-06-09 BR BR112021024855A patent/BR112021024855A2/pt unknown
- 2020-06-09 CA CA3142932A patent/CA3142932A1/en active Pending
- 2020-06-09 CN CN202080054069.6A patent/CN114502575A/zh active Pending
- 2020-06-09 JP JP2021573242A patent/JP2022536338A/ja active Pending
- 2020-06-09 TW TW109119333A patent/TW202112807A/zh unknown
- 2020-06-09 AU AU2020292256A patent/AU2020292256B2/en active Active
- 2020-06-09 EP EP20821636.6A patent/EP3980447A4/en not_active Withdrawn
- 2020-06-09 WO PCT/US2020/036846 patent/WO2020251954A1/en unknown
- 2020-06-09 PE PE2021002049A patent/PE20220233A1/es unknown
-
2021
- 2021-12-09 IL IL288863A patent/IL288863A/en unknown
- 2021-12-10 CL CL2021003295A patent/CL2021003295A1/es unknown
- 2021-12-10 US US17/643,631 patent/US20220204991A1/en active Pending
- 2021-12-10 CO CONC2021/0016797A patent/CO2021016797A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
WO2020251954A1 (en) | 2020-12-17 |
AU2020292256A1 (en) | 2022-01-06 |
EP3980447A4 (en) | 2023-07-26 |
CN114502575A (zh) | 2022-05-13 |
IL288863A (en) | 2022-02-01 |
AU2020292256B2 (en) | 2023-01-19 |
CA3142932A1 (en) | 2020-12-17 |
BR112021024855A2 (pt) | 2022-05-03 |
PE20220233A1 (es) | 2022-02-07 |
US20220204991A1 (en) | 2022-06-30 |
EP3980447A1 (en) | 2022-04-13 |
CO2021016797A2 (es) | 2022-01-17 |
KR20220035107A (ko) | 2022-03-21 |
TW202112807A (zh) | 2021-04-01 |
JP2022536338A (ja) | 2022-08-15 |
MX2021015076A (es) | 2022-06-02 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CL2021003295A1 (es) | Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas. | |
CO2019000395A2 (es) | Genes de mini–distrofina optimizados y casetes de expresión y su uso | |
PE20220231A1 (es) | Proteinas de fusion flt3l-fc y metodos de uso | |
AR122050A2 (es) | Proteínas de fusión, bacterias recombinantes y métodos de uso de las bacterias recombinantes | |
CL2017002731A1 (es) | Adenovirus oncolítico que codifica una proteína b7 | |
MX2020004092A (es) | Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas. | |
CL2017002199A1 (es) | Vectores retrovirales a lentivirales | |
CL2020001992A1 (es) | Composiciones de virus adeno-asociadas para la transferencia de genes pah y métodos de uso de los mismos | |
CO2021006362A2 (es) | Construcciones de ácido nucleico y métodos de uso | |
CL2020001451A1 (es) | Proteínas modificadas de unión a adn. | |
CO2021011198A2 (es) | Receptores de citocinas quiméricos constitutivamente activos | |
DOP2016000220A (es) | PROTEÍNAS DE FUSIÓN TATk-CDKL5, COMPOSICIONES, FORMULACIONES Y USO DE ESTAS. | |
CO2020016147A2 (es) | Composiciones de virus adenoasociado (aav) | |
EA201791918A1 (ru) | Модификация белков клеток-хозяев | |
BR112022010179A2 (pt) | Anticorpos anti-vírus da febre amarela, e métodos de sua geração e uso | |
AU2020299026A8 (en) | Methods and AAV vectors for in vivo transduction | |
CO2020010793A2 (es) | Composiciones de virus adeno-asociado para restaurar la función del gen pah y métodos de uso de las mismas | |
CO2022018370A2 (es) | Composiciones de virus adenoasociados para restaurar la función del gen pah y los métodos de uso de estas | |
AR119266A1 (es) | Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas | |
CL2023001860A1 (es) | Composiciones y métodos para reducir hla-a en una célula | |
EA201992405A1 (ru) | Система управления | |
CO2022010422A2 (es) | Anticuerpo anti antígeno-4 asociado al linfocito t citotóxico (ctla-4) y uso del mismo | |
AR110871A1 (es) | Proteínas de fusión de factor ix y métodos de fabricación y uso de las mismas | |
AR122165A1 (es) | Composiciones de virus adenoasociados para restaurar la función del gen pah y los métodos de uso de estas | |
WO2024036250A3 (en) | Adeno-associated virus compositions for arsa gene transfer and methods of use thereof |