JP2024517737A5 - - Google Patents

Info

Publication number
JP2024517737A5
JP2024517737A5 JP2023566490A JP2023566490A JP2024517737A5 JP 2024517737 A5 JP2024517737 A5 JP 2024517737A5 JP 2023566490 A JP2023566490 A JP 2023566490A JP 2023566490 A JP2023566490 A JP 2023566490A JP 2024517737 A5 JP2024517737 A5 JP 2024517737A5
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
composition according
nucleic acid
ngly1
nucleotide sequence
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.)
Pending
Application number
JP2023566490A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024517737A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/025834 external-priority patent/WO2022231950A1/en
Publication of JP2024517737A publication Critical patent/JP2024517737A/ja
Publication of JP2024517737A5 publication Critical patent/JP2024517737A5/ja
Pending legal-status Critical Current

Links

JP2023566490A 2021-04-26 2022-04-21 Ｎｇｙｌ１欠損症を治療する組成物及び方法 Pending JP2024517737A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163180065P 2021-04-26 2021-04-26
US63/180,065 2021-04-26
PCT/US2022/025834 WO2022231950A1 (en) 2021-04-26 2022-04-21 Compositions and methods for treating ngyl1 deficiency

Publications (2)

Publication Number Publication Date
JP2024517737A JP2024517737A (ja) 2024-04-23
JP2024517737A5 true JP2024517737A5 (https=) 2025-06-02

Family

ID=83848571

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023566490A Pending JP2024517737A (ja) 2021-04-26 2022-04-21 Ｎｇｙｌ１欠損症を治療する組成物及び方法

Country Status (5)

Country Link
US (1) US20240218396A1 (https=)
EP (1) EP4329824A4 (https=)
JP (1) JP2024517737A (https=)
CA (1) CA3216809A1 (https=)
WO (1) WO2022231950A1 (https=)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
EP3564263A1 (en) * 2018-05-03 2019-11-06 Universitätsklinikum Hamburg-Eppendorf Fusion proteins comprising a cell surface marker specific vhh
WO2020210592A1 (en) * 2019-04-12 2020-10-15 University Of Massachusetts Recombinant aav gene therapy for ngyl1 deficiency

Similar Documents

Publication Publication Date Title
US20220331409A1 (en) Factor ix gene therapy
JP2020537544A5 (https=)
RU2021102893A (ru) Модифицированные гены атаксии фридрейха и векторы для генной терапии
US20240141377A1 (en) Controlled expression of viral proteins
CA2720097A1 (en) Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US12359219B2 (en) Transgene cassettes designed to express a human MECP2 gene
CN101511373A (zh) 用于基因治疗的修饰的因子viii和因子ix基因和载体
US20240318199A1 (en) Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
WO2021034222A9 (ru) Выделенный модифицированный белок vp1 капсида aav5
US20220364114A1 (en) Controlled expression of viral proteins
CA3100405A1 (en) Adeno-associated viral vector-mediated gene therapy for treating fragile x-associated disorders
JP2023537980A5 (https=)
JP2025539750A (ja) 前頭側頭型認知症の遺伝子治療
CN116096904A (zh) 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途
KR20250102136A (ko) 중추 신경계에서의 유전자 발현을 위한 핵산 조절 요소 및 사용 방법
JP2023059858A5 (https=)
JP7820422B2 (ja) ジスフェリン二重ベクターを用いた遺伝子治療
CN120548368A (zh) 用于正确包装重复元件的腺相关病毒载体
JP7854125B2 (ja) 新規なデュアルヘルパープラスミド
JP2023542241A5 (https=)
JP2024517737A5 (https=)
WO2025162449A1 (en) Compositions and methods for the treatment of heart disease
RU2820088C1 (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
US20260085328A1 (en) Controlled expression of viral proteins
JP2024546578A5 (https=)