CN114502575A - 用于arsa基因转移的腺相关病毒组合物和其使用方法 - Google Patents

用于arsa基因转移的腺相关病毒组合物和其使用方法 Download PDF

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Publication number
CN114502575A
CN114502575A CN202080054069.6A CN202080054069A CN114502575A CN 114502575 A CN114502575 A CN 114502575A CN 202080054069 A CN202080054069 A CN 202080054069A CN 114502575 A CN114502575 A CN 114502575A
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amino acid
seq
capsid protein
protein corresponding
gly
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CN202080054069.6A
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Chinese (zh)
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T.B.圣马丁
A.B.西摩尔
H.鲁宾
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Cognate3 LLC
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Cognate3 LLC
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    • 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
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    • 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
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    • 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)

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  • Health & Medical Sciences (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
CN202080054069.6A 2019-06-10 2020-06-09 用于arsa基因转移的腺相关病毒组合物和其使用方法 Pending CN114502575A (zh)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201962859539P 2019-06-10 2019-06-10
US62/859,539 2019-06-10
US201962866374P 2019-06-25 2019-06-25
US62/866,374 2019-06-25
US201962915523P 2019-10-15 2019-10-15
US62/915,523 2019-10-15
US202062960487P 2020-01-13 2020-01-13
US62/960,487 2020-01-13
US202062987858P 2020-03-10 2020-03-10
US62/987,858 2020-03-10
US202063010970P 2020-04-16 2020-04-16
US63/010,970 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
CN114502575A true CN114502575A (zh) 2022-05-13

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CN202080054069.6A Pending CN114502575A (zh) 2019-06-10 2020-06-09 用于arsa基因转移的腺相关病毒组合物和其使用方法

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US (1) US20220204991A1 (https=)
EP (1) EP3980447A4 (https=)
JP (1) JP2022536338A (https=)
KR (1) KR20220035107A (https=)
CN (1) CN114502575A (https=)
AU (1) AU2020292256B2 (https=)
BR (1) BR112021024855A2 (https=)
CA (1) CA3142932A1 (https=)
CL (1) CL2021003295A1 (https=)
CO (1) CO2021016797A2 (https=)
IL (1) IL288863A (https=)
MX (1) MX2021015076A (https=)
PE (1) PE20220233A1 (https=)
TW (1) TW202112807A (https=)
WO (1) WO2020251954A1 (https=)

Families Citing this family (4)

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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
AU2023273809A1 (en) * 2022-05-16 2025-01-02 Genzyme Corporation Methods of treating metachromatic leukodystrophy
JP2025525792A (ja) 2022-07-29 2025-08-07 リジェネロン・ファーマシューティカルズ・インコーポレイテッド トランスフェリン受容体１に対して再標的化されるウイルス粒子
WO2024036250A2 (en) * 2022-08-10 2024-02-15 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof

Citations (5)

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CN106661591A (zh) * 2014-05-02 2017-05-10 建新公司 用于视网膜及cns基因疗法的aav载体
CN107532177A (zh) * 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
CN107828820A (zh) * 2010-10-27 2018-03-23 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
CN108367055A (zh) * 2015-05-15 2018-08-03 明尼苏达大学董事会 用于治疗性递送到中枢神经系统的腺相关物

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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
TWI813532B (zh) * 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
KR102528641B1 (ko) * 2018-02-01 2023-05-03 호몰로지 메디슨, 인크. Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
US20220127625A1 (en) * 2019-02-15 2022-04-28 Generation Bio Co. Modulation of rep protein activity in closed-ended dna (cedna) production
BR112021021908A2 (pt) * 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107828820A (zh) * 2010-10-27 2018-03-23 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
CN106661591A (zh) * 2014-05-02 2017-05-10 建新公司 用于视网膜及cns基因疗法的aav载体
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
CN107532177A (zh) * 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
CN108367055A (zh) * 2015-05-15 2018-08-03 明尼苏达大学董事会 用于治疗性递送到中枢神经系统的腺相关物

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MX2021015076A (es) 2022-06-02
JP2022536338A (ja) 2022-08-15
WO2020251954A1 (en) 2020-12-17
EP3980447A1 (en) 2022-04-13
TW202112807A (zh) 2021-04-01
AU2020292256B2 (en) 2023-01-19
IL288863A (en) 2022-02-01
EP3980447A4 (en) 2023-07-26
AU2020292256A1 (en) 2022-01-06
PE20220233A1 (es) 2022-02-07
BR112021024855A2 (pt) 2022-05-03
KR20220035107A (ko) 2022-03-21
CL2021003295A1 (es) 2022-09-23
US20220204991A1 (en) 2022-06-30
CO2021016797A2 (es) 2022-01-17
CA3142932A1 (en) 2020-12-17

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