JP2024523445A5 - - Google Patents

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Publication number
JP2024523445A5
JP2024523445A5 JP2023578738A JP2023578738A JP2024523445A5 JP 2024523445 A5 JP2024523445 A5 JP 2024523445A5 JP 2023578738 A JP2023578738 A JP 2023578738A JP 2023578738 A JP2023578738 A JP 2023578738A JP 2024523445 A5 JP2024523445 A5 JP 2024523445A5
Authority
JP
Japan
Prior art keywords
nucleic acid
seq
pharmaceutical
sequence
aav
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
JP2023578738A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024523445A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2022/066922 external-priority patent/WO2022268835A1/en
Publication of JP2024523445A publication Critical patent/JP2024523445A/ja
Publication of JP2024523445A5 publication Critical patent/JP2024523445A5/ja
Pending legal-status Critical Current

Links

JP2023578738A 2021-06-21 2022-06-21 α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用 Pending JP2024523445A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP21180613.8 2021-06-21
EP21180613 2021-06-21
EP22168018 2022-04-12
EP22168018.4 2022-04-12
PCT/EP2022/066922 WO2022268835A1 (en) 2021-06-21 2022-06-21 Gene constructs for silencing alpha-synuclein and uses thereof

Publications (2)

Publication Number Publication Date
JP2024523445A JP2024523445A (ja) 2024-06-28
JP2024523445A5 true JP2024523445A5 (enExample) 2025-06-30

Family

ID=82358408

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023578738A Pending JP2024523445A (ja) 2021-06-21 2022-06-21 α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用

Country Status (4)

Country Link
US (1) US20250382611A1 (enExample)
EP (1) EP4359525A1 (enExample)
JP (1) JP2024523445A (enExample)
WO (1) WO2022268835A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025051805A1 (en) 2023-09-04 2025-03-13 Uniqure Biopharma B.V. Novel neurotropic recombinant adeno-associated virus particles
WO2025078602A1 (en) * 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Nucleic acid regulation of msh3 in repeat expansion disorders
AU2024357870A1 (en) * 2023-10-11 2026-04-23 Uniqure Biopharma B.V. Further novel systems for nucleic acid regulation

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005045034A2 (en) * 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA)
CA2580189C (en) * 2004-09-29 2013-05-21 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
DK2173888T3 (en) 2007-07-26 2016-11-28 Uniqure Ip Bv Baculovirus vectors comprising repeating coding sequences WITH differential preferred codons
US20090176729A1 (en) * 2007-12-14 2009-07-09 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
JP2011512156A (ja) 2008-02-19 2011-04-21 アムステルダム モレキュラー セラピューティクス ビー．ブイ． 昆虫細胞におけるパルボウイルスｒｅｐ及びｃａｐタンパク質の発現の最適化
US9255266B2 (en) * 2009-05-06 2016-02-09 Rutgers, The State University Of New Jersey RNA targeting in alpha-synucleinopathies
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
US8993532B2 (en) * 2010-04-23 2015-03-31 Cold Spring Harbor Laboratory Structurally designed shRNAs
CA2817960C (en) * 2010-11-17 2020-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
JP6198278B2 (ja) 2011-09-08 2017-09-20 ユニキュアー アイピー ビー．ブイ． Ａａｖ調製物からの混入ウイルスの除去
HRP20190992T1 (hr) 2014-12-24 2019-09-20 Uniqure Ip B.V. Suprimiranje gena za huntingtin inducirano s rnai
CN112501208A (zh) * 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
KR102931619B1 (ko) * 2018-08-03 2026-02-26 젠자임 코포레이션 알파-시뉴클레인에 대한 변이형 RNAi
WO2020104295A1 (en) * 2018-11-19 2020-05-28 Uniqure Ip B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
CA3119721A1 (en) * 2018-11-19 2020-05-28 Uniqure Ip B.V. A companion diagnostic to monitor the effects of gene therapy
AU2020395113A1 (en) * 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing

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