KR20220035107A - Arsa 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 - Google Patents

Arsa 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Download PDF

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KR20220035107A
KR20220035107A KR1020227000707A KR20227000707A KR20220035107A KR 20220035107 A KR20220035107 A KR 20220035107A KR 1020227000707 A KR1020227000707 A KR 1020227000707A KR 20227000707 A KR20227000707 A KR 20227000707A KR 20220035107 A KR20220035107 A KR 20220035107A
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amino acid
seq
capsid protein
protein corresponding
gly
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Korean (ko)
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마틴 티아 바보발 세인트
앨버트 반즈 세이무어
힐러드 루빈
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호몰로지 메디슨, 인크.
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12Y301/06001—Arylsulfatase (3.1.6.1)

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  • Biodiversity & Conservation Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020227000707A 2019-06-10 2020-06-09 Arsa 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Pending KR20220035107A (ko)

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
KR20220035107A true KR20220035107A (ko) 2022-03-21

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KR1020227000707A Pending KR20220035107A (ko) 2019-06-10 2020-06-09 Arsa 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법

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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)

* Cited by examiner, † Cited by third party
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
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
EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
EP3245221A4 (en) * 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
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
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

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Publication number Publication date
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
CL2021003295A1 (es) 2022-09-23
US20220204991A1 (en) 2022-06-30
CO2021016797A2 (es) 2022-01-17
CN114502575A (zh) 2022-05-13
CA3142932A1 (en) 2020-12-17

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