JP2024532464A5 - - Google Patents

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Publication number
JP2024532464A5
JP2024532464A5 JP2024513758A JP2024513758A JP2024532464A5 JP 2024532464 A5 JP2024532464 A5 JP 2024532464A5 JP 2024513758 A JP2024513758 A JP 2024513758A JP 2024513758 A JP2024513758 A JP 2024513758A JP 2024532464 A5 JP2024532464 A5 JP 2024532464A5
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JP
Japan
Prior art keywords
compound according
aforementioned
peg
pkkkrkv
formula
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
JP2024513758A
Other languages
English (en)
Japanese (ja)
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JP2024532464A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/075691 external-priority patent/WO2023034817A1/en
Publication of JP2024532464A publication Critical patent/JP2024532464A/ja
Publication of JP2024532464A5 publication Critical patent/JP2024532464A5/ja
Pending legal-status Critical Current

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JP2024513758A 2021-09-01 2022-08-30 デュシェンヌ型筋ジストロフィーにおいてエクソン44をスキッピングするための化合物及び方法 Pending JP2024532464A (ja)

Applications Claiming Priority (23)

Application Number Priority Date Filing Date Title
US202163239645P 2021-09-01 2021-09-01
US202163239671P 2021-09-01 2021-09-01
US63/239,671 2021-09-01
US63/239,645 2021-09-01
US202163290960P 2021-12-17 2021-12-17
US63/290,960 2021-12-17
US202163292685P 2021-12-22 2021-12-22
US63/292,685 2021-12-22
US202263298565P 2022-01-11 2022-01-11
US63/298,565 2022-01-11
US202263268577P 2022-02-25 2022-02-25
US202263268580P 2022-02-25 2022-02-25
US63/268,577 2022-02-25
US63/268,580 2022-02-25
US202263362294P 2022-03-31 2022-03-31
US63/362,294 2022-03-31
US202263362423P 2022-04-04 2022-04-04
US63/362,423 2022-04-04
US202263337560P 2022-05-02 2022-05-02
US63/337,560 2022-05-02
US202263354456P 2022-06-22 2022-06-22
US63/354,456 2022-06-22
PCT/US2022/075691 WO2023034817A1 (en) 2021-09-01 2022-08-30 Compounds and methods for skipping exon 44 in duchenne muscular dystrophy

Publications (2)

Publication Number Publication Date
JP2024532464A JP2024532464A (ja) 2024-09-05
JP2024532464A5 true JP2024532464A5 (enExample) 2025-09-08

Family

ID=83448049

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024513758A Pending JP2024532464A (ja) 2021-09-01 2022-08-30 デュシェンヌ型筋ジストロフィーにおいてエクソン44をスキッピングするための化合物及び方法

Country Status (9)

Country Link
US (1) US20260098262A9 (enExample)
EP (1) EP4395829A1 (enExample)
JP (1) JP2024532464A (enExample)
KR (1) KR20240082344A (enExample)
AU (1) AU2022339769A1 (enExample)
CA (1) CA3229661A1 (enExample)
IL (1) IL311139A (enExample)
MX (1) MX2024002640A (enExample)
WO (1) WO2023034817A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025038901A1 (en) * 2023-08-17 2025-02-20 Entrada Therapeutics, Inc. Cyclic peptides for delivering therapeutics
WO2025072246A1 (en) * 2023-09-26 2025-04-03 Entrada Therapeutics, Inc. Compounds and methods for skipping exon 50 in duchenne muscular dystrophy

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
DE69034150T2 (de) 1989-10-24 2005-08-25 Isis Pharmaceuticals, Inc., Carlsbad 2'-Modifizierte Oligonukleotide
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
EP0538194B1 (de) 1991-10-17 1997-06-04 Novartis AG Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
JPH08504559A (ja) 1992-12-14 1996-05-14 ハネウエル・インコーポレーテッド 個別に制御される冗長巻線を有するモータシステム
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
CA2159631A1 (en) 1993-03-30 1994-10-13 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
US5792747A (en) 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
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
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
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
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
JP2002535247A (ja) 1998-11-26 2002-10-22 ペンタファルム アクチェンゲゼルシャフト 輸送系複合体
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
EP1235914A2 (en) 1999-11-24 2002-09-04 Joseph Rosenecker Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells
US7034109B2 (en) 2000-10-13 2006-04-25 Christophe Bonny Intracellular delivery of biological effectors
US7033597B2 (en) 2000-10-13 2006-04-25 Université de Lausanne Intracellular delivery of biological effectors
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
JP6807831B2 (ja) * 2014-05-21 2021-01-06 エントラーダ セラピューティクス,インコーポレイテッド 細胞膜透過性ペプチド、並びにこの作製方法及び使用方法
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EP4058032A4 (en) * 2019-12-19 2024-01-10 Entrada Therapeutics, Inc. Compositions for delivery of antisense compounds
EP4314016A1 (en) * 2021-03-31 2024-02-07 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides

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