JPWO2022268835A5 - - Google Patents

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Publication number
JPWO2022268835A5
JPWO2022268835A5 JP2023578738A JP2023578738A JPWO2022268835A5 JP WO2022268835 A5 JPWO2022268835 A5 JP WO2022268835A5 JP 2023578738 A JP2023578738 A JP 2023578738A JP 2023578738 A JP2023578738 A JP 2023578738A JP WO2022268835 A5 JPWO2022268835 A5 JP WO2022268835A5
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JP
Japan
Prior art keywords
nucleic acid
medicament
vehicle
seq
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
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English (en)
Japanese (ja)
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JP2024523445A (ja
JP2024523445A5 (https=
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
Publication of JPWO2022268835A5 publication Critical patent/JPWO2022268835A5/ja
Pending legal-status Critical Current

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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 (3)

Publication Number Publication Date
JP2024523445A JP2024523445A (ja) 2024-06-28
JP2024523445A5 JP2024523445A5 (https=) 2025-06-30
JPWO2022268835A5 true JPWO2022268835A5 (https=) 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 (https=)
EP (1) EP4359525A1 (https=)
JP (1) JP2024523445A (https=)
WO (1) WO2022268835A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024337260A1 (en) 2023-09-04 2026-03-12 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)
WO2006039253A2 (en) * 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
SI3093345T1 (sl) 2007-07-26 2019-08-30 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami
WO2009079399A2 (en) * 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
EP3257937B1 (en) 2008-02-19 2022-08-03 uniQure IP B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
WO2010129791A1 (en) * 2009-05-06 2010-11-11 University Of Medicine And Dentistry 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
HRP20182039T1 (hr) * 2010-04-23 2019-02-08 Cold Spring Harbor Laboratory NOVE STRUKTURNO DIZAJNIRANE MOLEKULE shRNA
CA3077910A1 (en) * 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
PT2744895E (pt) 2011-09-08 2016-02-08 Uniqure Ip Bv Remoção de vírus contaminantes de preparações de aav
AU2015370903B2 (en) 2014-12-24 2021-06-17 Uniqure Ip B.V. RNAi induced huntingtin gene suppression
BR112020006671A2 (pt) * 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
EP3830265A1 (en) * 2018-08-03 2021-06-09 Genzyme Corporation Variant rnai against alpha-synuclein
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
WO2021113270A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing

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