JPWO2022271818A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2022271818A5
JPWO2022271818A5 JP2023579457A JP2023579457A JPWO2022271818A5 JP WO2022271818 A5 JPWO2022271818 A5 JP WO2022271818A5 JP 2023579457 A JP2023579457 A JP 2023579457A JP 2023579457 A JP2023579457 A JP 2023579457A JP WO2022271818 A5 JPWO2022271818 A5 JP WO2022271818A5
Authority
JP
Japan
Prior art keywords
cag cag
compound
peptide
exocyclic
pmo
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
JP2023579457A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024524291A5 (https=
JP2024524291A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/034517 external-priority patent/WO2022271818A1/en
Publication of JP2024524291A publication Critical patent/JP2024524291A/ja
Publication of JP2024524291A5 publication Critical patent/JP2024524291A5/ja
Publication of JPWO2022271818A5 publication Critical patent/JPWO2022271818A5/ja
Pending legal-status Critical Current

Links

JP2023579457A 2021-06-23 2022-06-22 Cugリピートを標的とするためのアンチセンス化合物及び方法 Pending JP2024524291A (ja)

Applications Claiming Priority (31)

Application Number Priority Date Filing Date Title
US202163213900P 2021-06-23 2021-06-23
US63/213,900 2021-06-23
US202163239671P 2021-09-01 2021-09-01
US202163239847P 2021-09-01 2021-09-01
US63/239,847 2021-09-01
US63/239,671 2021-09-01
US202163290892P 2021-12-17 2021-12-17
US202163290960P 2021-12-17 2021-12-17
US63/290,892 2021-12-17
US63/290,960 2021-12-17
US202263298565P 2022-01-11 2022-01-11
US63/298,565 2022-01-11
US202263305071P 2022-01-31 2022-01-31
US63/305,071 2022-01-31
US202263268577P 2022-02-25 2022-02-25
US63/268,577 2022-02-25
US202263314369P 2022-02-26 2022-02-26
US63/314,369 2022-02-26
US202263316634P 2022-03-04 2022-03-04
US63/316,634 2022-03-04
US202263317856P 2022-03-08 2022-03-08
US63/317,856 2022-03-08
US202263326201P 2022-03-31 2022-03-31
US202263362295P 2022-03-31 2022-03-31
US63/362,295 2022-03-31
US63/326,201 2022-03-31
US202263327179P 2022-04-04 2022-04-04
US63/327,179 2022-04-04
US202263339250P 2022-05-06 2022-05-06
US63/339,250 2022-05-06
PCT/US2022/034517 WO2022271818A1 (en) 2021-06-23 2022-06-22 Antisense compounds and methods for targeting cug repeats

Publications (3)

Publication Number Publication Date
JP2024524291A JP2024524291A (ja) 2024-07-05
JP2024524291A5 JP2024524291A5 (https=) 2025-07-01
JPWO2022271818A5 true JPWO2022271818A5 (https=) 2025-07-01

Family

ID=82608300

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023579457A Pending JP2024524291A (ja) 2021-06-23 2022-06-22 Cugリピートを標的とするためのアンチセンス化合物及び方法

Country Status (15)

Country Link
EP (1) EP4359006A1 (https=)
JP (1) JP2024524291A (https=)
KR (1) KR20240038967A (https=)
AU (1) AU2022298774A1 (https=)
CA (1) CA3222824A1 (https=)
CL (1) CL2023003840A1 (https=)
CO (1) CO2023018002A2 (https=)
DO (1) DOP2023000279A (https=)
EC (1) ECSP23096162A (https=)
GE (1) GEAP202416440A (https=)
IL (1) IL309001A (https=)
JO (1) JOP20230327A1 (https=)
MX (1) MX2023015509A (https=)
PE (1) PE20242357A1 (https=)
WO (1) WO2022271818A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4522221A1 (en) * 2022-05-09 2025-03-19 Entrada Therapeutics, Inc. Compositions and methods for delivery of nucleic acid therapeutics
GB2628421A (en) * 2023-03-24 2024-09-25 Syntherix Ltd Peptides and uses thereof
WO2024263649A1 (en) * 2023-06-19 2024-12-26 Yale University Methods and compositions for enrichment and sequencing of expansion-specific rna transcripts
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
WO2025137623A1 (en) * 2023-12-22 2025-06-26 Iris Medicine, Inc. Methods and compositions for treating ctg repeat expansion diseases
EP4692348A1 (en) * 2024-08-07 2026-02-11 Nocturna Therapeutics SL Artificial rnas for treating repeat expansion disorders

Family Cites Families (85)

* 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
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
AU637800B2 (en) 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
ATE269870T1 (de) 1989-10-24 2004-07-15 Isis Pharmaceuticals Inc 2'-modifizierte oligonukleotide
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
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.
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. 修飾リボザイム
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
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
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
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
WO1994002595A1 (en) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
EP0673559A1 (en) 1992-12-14 1995-09-27 Honeywell Inc. Motor system with individually controlled redundant windings
AU6449394A (en) 1993-03-30 1994-10-24 Sterling Winthrop Inc. 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
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
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
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
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5792747A (en) 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
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
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
EP1133317A1 (de) 1998-11-26 2001-09-19 Pentapharm AG Transportsystemkonjugate
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma 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
CA2392490A1 (en) 1999-11-24 2001-05-31 Mcs Micro Carrier Systems Gmbh Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US6960648B2 (en) 2000-10-13 2005-11-01 Universite De Lausanne 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
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
US7523096B2 (en) 2003-12-03 2009-04-21 Google Inc. Methods and systems for personalized network searching
AU2005252663B2 (en) 2004-06-03 2011-07-07 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
NZ563206A (en) 2005-04-22 2009-09-25 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mRNA by interfering with the binding or SR proteins and by interfering with secondary RNA structure
JP5342881B2 (ja) 2006-01-27 2013-11-13 アイシス ファーマシューティカルズ, インコーポレーテッド 6−修飾された二環式核酸類似体
EP1986697B1 (en) 2006-02-17 2016-06-29 GE Healthcare Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
WO2008036841A2 (en) 2006-09-22 2008-03-27 Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
WO2008049085A1 (en) 2006-10-18 2008-04-24 Isis Pharmaceuticals, Inc. Antisense compounds
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102240217B1 (ko) * 2012-09-25 2021-04-14 젠자임 코포레이션 근긴장성 이영양증의 치료용 펩티드-결합 모르폴리노 안티센스 올리고뉴클레오티드
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
CN105121648B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CA2894684A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
MX2016007325A (es) 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
US20180344817A1 (en) 2015-05-01 2018-12-06 Precision Biosciences, Inc. Precise deletion of chromosomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases
EP3359667A1 (en) 2015-10-05 2018-08-15 ProQR Therapeutics II B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
JP6657520B2 (ja) 2016-02-03 2020-03-04 株式会社神戸製鋼所 タイヤ試験装置のタイヤ空気充填機構及びタイヤ空気充填方法
KR20170130253A (ko) 2016-05-18 2017-11-28 에스케이텔레콤 주식회사 적응형 스트리밍 서비스 제공 방법 및 이를 위한 장치
US10265249B2 (en) 2016-09-29 2019-04-23 The Procter & Gamble Company Fibrous structures comprising glyceride copolymers
WO2018093990A1 (en) 2016-11-16 2018-05-24 The Regents Of The University Of California Inhibitors of crispr-cas9
KR20180102871A (ko) 2017-03-08 2018-09-18 엘지전자 주식회사 이동단말기 및 이동단말기의 차량 제어 방법
WO2018165541A1 (en) 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
TW201945014A (zh) * 2018-02-22 2019-12-01 美商安卓達治療股份有限公司 用於治療粒線體性神經胃腸腦病變之組合物及方法
GB201812972D0 (en) * 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
US20230020092A1 (en) * 2019-12-19 2023-01-19 Entrada Therapeutics, Inc. Compositions for delivery of antisense compounds

Similar Documents

Publication Publication Date Title
JP7060216B2 (ja) 多様な選択された臓器又は組織をターゲティングするための物質
JP7430713B2 (ja) 核酸送達のための脂質化カチオン性ペプチド化合物を含む脂質ナノ粒子製剤
CN103998458B (zh) 具有中央疏水域的细胞穿透肽
US8324365B2 (en) Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US8575305B2 (en) Cell penetrating peptides
CN103747805B (zh) 用于治疗、延缓和/或预防人类遗传性疾病如强直性肌营养不良症1型(dm1)的化合物
TW202346591A (zh) 連結有載體胜肽的核酸
JP2010533170A (ja) 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子
CN101815535A (zh) 用于使化合物靶向多种选定器官或组织的分子
CA2498772A1 (en) Modified pna molecules
IL309001A (en) Antisense compounds and methods for targeting CUG repeats
CN114615998A (zh) 缀合物及其用途
AU2022337260A1 (en) Compositions and methods for skipping exon 45 in duchenne muscular dystrophy
KR102487942B1 (ko) 펩티드 담체 상의 다중 올리고뉴클레오티드 모이어티
JPWO2022271818A5 (https=)
US20040072743A1 (en) Pharmaceutical composition of modified pna molecules
Chiarantini et al. Comparison of novel delivery systems for antisense peptide nucleic acids
JP2003511466A (ja) 修飾pna分子
JPWO2023034818A5 (https=)
HUP0200679A2 (en) Fragments of virus protein 2 or 3 of the polyoma virus, used for transporting active ingredients
CN101495505A (zh) 被膦酸酯或膦酸取代的手性化合物
WO2002053574A2 (en) Modified pna molecules
WO2022261029A2 (en) Peptide nucleic acid therapeutics for trinucleotide repeat disorders
WO2022025148A1 (ja) miR-96-5pインヒビターとそれを含有する医薬組成物
HK40075520A (zh) 缀合物及其用途