JP2022531861A5 - - Google Patents

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Publication number
JP2022531861A5
JP2022531861A5 JP2021564955A JP2021564955A JP2022531861A5 JP 2022531861 A5 JP2022531861 A5 JP 2022531861A5 JP 2021564955 A JP2021564955 A JP 2021564955A JP 2021564955 A JP2021564955 A JP 2021564955A JP 2022531861 A5 JP2022531861 A5 JP 2022531861A5
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JP
Japan
Prior art keywords
sequence
vector
raav
harsa
pharmaceutical composition
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2021564955A
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English (en)
Japanese (ja)
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JP2022531861A (ja
JP7715640B2 (ja
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Priority claimed from PCT/US2020/031207 external-priority patent/WO2020227166A1/en
Publication of JP2022531861A publication Critical patent/JP2022531861A/ja
Publication of JP2022531861A5 publication Critical patent/JP2022531861A5/ja
Application granted granted Critical
Publication of JP7715640B2 publication Critical patent/JP7715640B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021564955A 2019-05-03 2020-05-02 異染性白質ジストロフィーの治療に有用な組成物 Active JP7715640B2 (ja)

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 (3)

Publication Number Publication Date
JP2022531861A JP2022531861A (ja) 2022-07-12
JP2022531861A5 true JP2022531861A5 (https=) 2023-05-12
JP7715640B2 JP7715640B2 (ja) 2025-07-30

Family

ID=73050896

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021564955A Active JP7715640B2 (ja) 2019-05-03 2020-05-02 異染性白質ジストロフィーの治療に有用な組成物

Country Status (17)

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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WO2020251954A1 (en) * 2019-06-10 2020-12-17 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
MX2022014258A (es) * 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
IL307633A (en) * 2021-04-12 2023-12-01 Univ Pennsylvania Useful preparations in the treatment of spinal muscular atrophy (SBMA)
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
US20240269328A1 (en) * 2021-06-08 2024-08-15 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof
WO2023133584A1 (en) * 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
EP4504953A4 (en) * 2022-04-05 2026-04-15 Astellas Gene Therapies Inc DOSAGE OF ADENE-ASSOCIATED VIRUS AND ITS USES
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
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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