KR20220020261A - 이염성 백질이영양증의 치료에 유용한 조성물 - Google Patents

이염성 백질이영양증의 치료에 유용한 조성물 Download PDF

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KR20220020261A
KR20220020261A KR1020217039381A KR20217039381A KR20220020261A KR 20220020261 A KR20220020261 A KR 20220020261A KR 1020217039381 A KR1020217039381 A KR 1020217039381A KR 20217039381 A KR20217039381 A KR 20217039381A KR 20220020261 A KR20220020261 A KR 20220020261A
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vector
raav
seq
sequence
certain embodiments
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Korean (ko)
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줄리에트 호르도
제임스 윌슨
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더 트러스티스 오브 더 유니버시티 오브 펜실베니아
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Publication of KR20220020261A publication Critical patent/KR20220020261A/ko
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KR1020217039381A 2019-05-03 2020-05-02 이염성 백질이영양증의 치료에 유용한 조성물 Pending KR20220020261A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962843091P 2019-05-03 2019-05-03
US62/843,091 2019-05-03
PCT/US2020/031207 WO2020227166A1 (en) 2019-05-03 2020-05-02 Compositions useful in treatment of metachromatic leukodystrophy

Publications (1)

Publication Number Publication Date
KR20220020261A true KR20220020261A (ko) 2022-02-18

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KR1020217039381A Pending KR20220020261A (ko) 2019-05-03 2020-05-02 이염성 백질이영양증의 치료에 유용한 조성물

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Country Link
US (2) US12338451B2 (https=)
EP (1) EP3962454A4 (https=)
JP (1) JP7715640B2 (https=)
KR (1) KR20220020261A (https=)
CN (1) CN114072129A (https=)
AR (1) AR118850A1 (https=)
AU (1) AU2020270421A1 (https=)
BR (1) BR112021021908A2 (https=)
CA (1) CA3135539A1 (https=)
CL (1) CL2021002881A1 (https=)
CO (1) CO2021016323A2 (https=)
IL (1) IL287715A (https=)
MX (1) MX2021013420A (https=)
PE (1) PE20220930A1 (https=)
SG (1) SG11202111867XA (https=)
TW (1) TW202108760A (https=)
WO (1) WO2020227166A1 (https=)

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US20230304034A1 (en) * 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
KR20230170022A (ko) * 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
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
IL308787A (en) * 2021-06-08 2024-01-01 Univ Pennsylvania Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses
US20250177495A1 (en) * 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
WO2023196863A1 (en) * 2022-04-05 2023-10-12 Astellas Gene Therapies, Inc. Adeno-associated virus potency assay and uses thereof
AU2023273809A1 (en) * 2022-05-16 2025-01-02 Genzyme Corporation Methods of treating metachromatic leukodystrophy
WO2024036250A2 (en) * 2022-08-10 2024-02-15 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
WO2024079361A1 (en) * 2022-10-14 2024-04-18 Neuway Pharma Gmbh A PROTEIN- OR PEPTIDE-BASED CAPSULE (PPC), PREFERABLY A VLP, LOADED WITH A MESSENGER RNA (mRNA) AND A METHOD OF PRODUCTION AND PURIFICATION THEREOF
CN118389675B (zh) * 2024-05-27 2025-06-27 南方医科大学珠江医院 芳香族氨基酸脱羧酶作为标志物在肝性脑病的检测产品中的应用

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