PE20220233A1 - Composiciones de virus adenoasociados para la transferencia del gen de arsa y metodos de uso de las mismas - Google Patents
Composiciones de virus adenoasociados para la transferencia del gen de arsa y metodos de uso de las mismasInfo
- Publication number
- PE20220233A1 PE20220233A1 PE2021002049A PE2021002049A PE20220233A1 PE 20220233 A1 PE20220233 A1 PE 20220233A1 PE 2021002049 A PE2021002049 A PE 2021002049A PE 2021002049 A PE2021002049 A PE 2021002049A PE 20220233 A1 PE20220233 A1 PE 20220233A1
- Authority
- PE
- Peru
- Prior art keywords
- compositions
- adeno
- methods
- gene transfer
- associated viruses
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 4
- 101150007653 Arsa gene Proteins 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 241000700605 Viruses Species 0.000 title 1
- 102100022146 Arylsulfatase A Human genes 0.000 abstract 2
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 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)
Applications Claiming Priority (7)
| 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 | |
| PCT/US2020/036846 WO2020251954A1 (en) | 2019-06-10 | 2020-06-09 | Adeno-associated virus compositions for arsa gene transfer and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20220233A1 true PE20220233A1 (es) | 2022-02-07 |
Family
ID=73782242
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2021002049A PE20220233A1 (es) | 2019-06-10 | 2020-06-09 | Composiciones de virus adenoasociados para la transferencia del gen de arsa y metodos de uso de las mismas |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20220204991A1 (enExample) |
| EP (1) | EP3980447A4 (enExample) |
| JP (1) | JP2022536338A (enExample) |
| KR (1) | KR20220035107A (enExample) |
| CN (1) | CN114502575A (enExample) |
| AU (1) | AU2020292256B2 (enExample) |
| BR (1) | BR112021024855A2 (enExample) |
| CA (1) | CA3142932A1 (enExample) |
| CL (1) | CL2021003295A1 (enExample) |
| CO (1) | CO2021016797A2 (enExample) |
| IL (1) | IL288863A (enExample) |
| MX (1) | MX2021015076A (enExample) |
| PE (1) | PE20220233A1 (enExample) |
| TW (1) | TW202112807A (enExample) |
| WO (1) | WO2020251954A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2769577C1 (ru) * | 2021-06-01 | 2022-04-04 | федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) | Препарат для лечения метахроматической лейкодистрофии и способ ее лечения |
| 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 |
| CA3263388A1 (en) | 2022-07-29 | 2024-02-01 | Regeneron Pharmaceuticals, Inc. | VIRAL PARTICLES RETRIBUTED 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 (10)
| 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 |
| WO2012057363A1 (ja) * | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| LT3137497T (lt) * | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| MX2017009336A (es) * | 2015-01-16 | 2017-11-15 | Voyager Therapeutics Inc | Polinucleótidos dirigidos al sistema nervioso central. |
| JP6836999B2 (ja) * | 2015-03-24 | 2021-03-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ | アデノ随伴ウイルス変異体及びその使用方法 |
| HK1256341A1 (zh) * | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| TWI906646B (zh) * | 2015-06-18 | 2025-12-01 | 美商博得學院股份有限公司 | 降低脫靶效應的ｃｒｉｓｐｒ酶突變 |
| KR20230065367A (ko) * | 2018-02-01 | 2023-05-11 | 호몰로지 메디슨, 인크. | Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 |
| CN113454232B (zh) * | 2019-02-15 | 2024-10-29 | 世代生物公司 | 封闭端dna（cedna）产生中对rep蛋白活性的调节 |
| KR20220020261A (ko) * | 2019-05-03 | 2022-02-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 이염성 백질이영양증의 치료에 유용한 조성물 |
-
2020
- 2020-06-09 WO PCT/US2020/036846 patent/WO2020251954A1/en not_active Ceased
- 2020-06-09 BR BR112021024855A patent/BR112021024855A2/pt not_active IP Right Cessation
- 2020-06-09 EP EP20821636.6A patent/EP3980447A4/en not_active Withdrawn
- 2020-06-09 KR KR1020227000707A patent/KR20220035107A/ko not_active Ceased
- 2020-06-09 MX MX2021015076A patent/MX2021015076A/es unknown
- 2020-06-09 AU AU2020292256A patent/AU2020292256B2/en not_active Ceased
- 2020-06-09 JP JP2021573242A patent/JP2022536338A/ja active Pending
- 2020-06-09 PE PE2021002049A patent/PE20220233A1/es unknown
- 2020-06-09 CA CA3142932A patent/CA3142932A1/en active Pending
- 2020-06-09 TW TW109119333A patent/TW202112807A/zh unknown
- 2020-06-09 CN CN202080054069.6A patent/CN114502575A/zh active Pending
-
2021
- 2021-12-09 IL IL288863A patent/IL288863A/en unknown
- 2021-12-10 US US17/643,631 patent/US20220204991A1/en not_active Abandoned
- 2021-12-10 CL CL2021003295A patent/CL2021003295A1/es unknown
- 2021-12-10 CO CONC2021/0016797A patent/CO2021016797A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2020292256B2 (en) | 2023-01-19 |
| CA3142932A1 (en) | 2020-12-17 |
| JP2022536338A (ja) | 2022-08-15 |
| EP3980447A4 (en) | 2023-07-26 |
| BR112021024855A2 (pt) | 2022-05-03 |
| US20220204991A1 (en) | 2022-06-30 |
| AU2020292256A1 (en) | 2022-01-06 |
| WO2020251954A1 (en) | 2020-12-17 |
| EP3980447A1 (en) | 2022-04-13 |
| IL288863A (en) | 2022-02-01 |
| TW202112807A (zh) | 2021-04-01 |
| CO2021016797A2 (es) | 2022-01-17 |
| CL2021003295A1 (es) | 2022-09-23 |
| KR20220035107A (ko) | 2022-03-21 |
| MX2021015076A (es) | 2022-06-02 |
| CN114502575A (zh) | 2022-05-13 |
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