JP2024536132A5 - - Google Patents

Info

Publication number
JP2024536132A5
JP2024536132A5 JP2024519267A JP2024519267A JP2024536132A5 JP 2024536132 A5 JP2024536132 A5 JP 2024536132A5 JP 2024519267 A JP2024519267 A JP 2024519267A JP 2024519267 A JP2024519267 A JP 2024519267A JP 2024536132 A5 JP2024536132 A5 JP 2024536132A5
Authority
JP
Japan
Prior art keywords
antisense oligonucleotide
disease
related protein
polyq
protein
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
JP2024519267A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024536132A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2022/076748 external-priority patent/WO2023052317A1/en
Publication of JP2024536132A publication Critical patent/JP2024536132A/ja
Publication of JP2024536132A5 publication Critical patent/JP2024536132A5/ja
Pending legal-status Critical Current

Links

JP2024519267A 2021-09-29 2022-09-27 Rna編集方法 Pending JP2024536132A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21199761.4 2021-09-29
EP21199761 2021-09-29
PCT/EP2022/076748 WO2023052317A1 (en) 2021-09-29 2022-09-27 Rna editing

Publications (2)

Publication Number Publication Date
JP2024536132A JP2024536132A (ja) 2024-10-04
JP2024536132A5 true JP2024536132A5 (https=) 2025-10-02

Family

ID=78179173

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024519267A Pending JP2024536132A (ja) 2021-09-29 2022-09-27 Rna編集方法

Country Status (7)

Country Link
US (1) US20240336919A1 (https=)
EP (1) EP4408999A1 (https=)
JP (1) JP2024536132A (https=)
CN (1) CN118019848A (https=)
AR (1) AR127158A1 (https=)
TW (1) TW202329983A (https=)
WO (1) WO2023052317A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
GB202405143D0 (en) * 2024-04-11 2024-05-29 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of poly-q disease

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ/エス オリゴヌクレオチド類似体
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
WO2004046160A2 (en) 2002-11-18 2004-06-03 Santaris Pharma A/S Amino-lna, thio-lna and alpha-l-oxy-ln
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
WO2009099326A1 (en) * 2008-02-08 2009-08-13 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
DK2356129T3 (da) 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
US10202599B2 (en) 2011-08-11 2019-02-12 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2013033223A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
US20150315595A1 (en) 2012-03-12 2015-11-05 Santaris Pharma A/S Compositions and Methods for Modulation of ATXN3 Expression
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
LT2841578T (lt) * 2012-04-23 2017-09-25 Biomarin Technologies B.V. Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui
US10435430B2 (en) 2013-07-31 2019-10-08 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
EP3234134B1 (en) 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Targeted rna editing
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
WO2019158475A1 (en) 2018-02-14 2019-08-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
US20200385713A1 (en) * 2019-01-22 2020-12-10 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof

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