CN116437968A - 用于神经退行性病症的基因疗法 - Google Patents

用于神经退行性病症的基因疗法 Download PDF

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Publication number
CN116437968A
CN116437968A CN202180068282.7A CN202180068282A CN116437968A CN 116437968 A CN116437968 A CN 116437968A CN 202180068282 A CN202180068282 A CN 202180068282A CN 116437968 A CN116437968 A CN 116437968A
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raav
dose
day
administration
protein
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Chinese (zh)
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阿萨·阿贝利奥维奇
杰弗里·思维格尼
特拉维斯·刘易斯
奥尔加·乌斯片斯卡亚
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Privelle Therapeutics
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Privelle Therapeutics
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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CN202180068282.7A 2020-08-10 2021-08-10 用于神经退行性病症的基因疗法 Pending CN116437968A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063063852P 2020-08-10 2020-08-10
US63/063,852 2020-08-10
PCT/US2021/045443 WO2022035900A1 (en) 2020-08-10 2021-08-10 Gene therapies for neurodegenerative disorders

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CN116437968A true CN116437968A (zh) 2023-07-14

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US (1) US20230346979A1 (https=)
EP (1) EP4192517A1 (https=)
JP (1) JP2023537904A (https=)
KR (1) KR20230066360A (https=)
CN (1) CN116437968A (https=)
AU (1) AU2021325891A1 (https=)
CA (1) CA3190864A1 (https=)
IL (1) IL300409A (https=)
MX (1) MX2023001564A (https=)
WO (1) WO2022035900A1 (https=)

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KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
PL3953377T3 (pl) 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych
CN116672341A (zh) * 2022-02-23 2023-09-01 中国科学院上海药物研究所 雷帕霉素在治疗渐冻症和额颞叶痴呆中的应用
EP4598940A1 (en) 2022-10-04 2025-08-13 Eli Lilly and Company Gene therapy for trem2-associated diseases and disorders
EP4615981A1 (en) 2022-11-11 2025-09-17 Eli Lilly and Company Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same
KR20250131772A (ko) * 2022-11-13 2025-09-03 알렉시온 파마 인터내셔널 오퍼레이션즈 리미티드 전두측두엽 치매에 대한 유전자 요법
WO2024163823A1 (en) * 2023-02-02 2024-08-08 Shape Therapeutics Inc. Tissue-specific enhancers for regulating transcription
AU2024237478A1 (en) * 2023-03-16 2025-10-30 Genzyme Corporation Treatment of dry age-related macular degeneration
WO2025251041A1 (en) * 2024-05-31 2025-12-04 Bluerock Therapeutics Lp METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS
WO2026043765A1 (en) 2024-08-21 2026-02-26 Eli Lilly And Company Engineered regulatory splice elements for gene therapies

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ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
CN107002096A (zh) * 2014-10-06 2017-08-01 阿罗根有限公司 基于aav的基因疗法
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
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
KR20230079511A (ko) 2016-04-21 2023-06-07 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물
KR102719222B1 (ko) * 2017-05-11 2024-10-21 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 신경 세로이드 지질갈색소증에 대한 유전자 요법
MA50016A (fr) * 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
EP3692075A4 (en) 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
KR102709597B1 (ko) * 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
PL3953377T3 (pl) 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych

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JP2023537904A (ja) 2023-09-06
EP4192517A1 (en) 2023-06-14
WO2022035900A1 (en) 2022-02-17
US20230346979A1 (en) 2023-11-02
AU2021325891A1 (en) 2023-04-06
KR20230066360A (ko) 2023-05-15
CA3190864A1 (en) 2022-02-17
IL300409A (en) 2023-04-01
MX2023001564A (es) 2023-05-03

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