JP2020503010A5 - - Google Patents

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JP2020503010A5
JP2020503010A5 JP2019531379A JP2019531379A JP2020503010A5 JP 2020503010 A5 JP2020503010 A5 JP 2020503010A5 JP 2019531379 A JP2019531379 A JP 2019531379A JP 2019531379 A JP2019531379 A JP 2019531379A JP 2020503010 A5 JP2020503010 A5 JP 2020503010A5
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composition
antisense oligomer
oligomer conjugate
exon
administered
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JP2019531379A
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JP7125940B2 (ja
JP2020503010A (ja
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Priority claimed from PCT/US2017/066351 external-priority patent/WO2018118627A1/en
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Priority to JP2022075930A priority Critical patent/JP2022101695A/ja
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Publication of JP7125940B2 publication Critical patent/JP7125940B2/ja
Priority to JP2024095058A priority patent/JP7728923B2/ja
Priority to JP2025134907A priority patent/JP2025166161A/ja
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JP2019531379A 2016-12-19 2017-12-14 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート Active JP7125940B2 (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2022075930A JP2022101695A (ja) 2016-12-19 2022-05-02 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2024095058A JP7728923B2 (ja) 2016-12-19 2024-06-12 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2025134907A JP2025166161A (ja) 2016-12-19 2025-08-13 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662436199P 2016-12-19 2016-12-19
US62/436,199 2016-12-19
US201762443481P 2017-01-06 2017-01-06
US62/443,481 2017-01-06
US201762479177P 2017-03-30 2017-03-30
US62/479,177 2017-03-30
US201762562119P 2017-09-22 2017-09-22
US62/562,119 2017-09-22
PCT/US2017/066351 WO2018118627A1 (en) 2016-12-19 2017-12-14 Exon skipping oligomer conjugates for muscular dystrophy

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JP2022075930A Division JP2022101695A (ja) 2016-12-19 2022-05-02 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

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JP2020503010A JP2020503010A (ja) 2020-01-30
JP2020503010A5 true JP2020503010A5 (enExample) 2021-01-28
JP7125940B2 JP7125940B2 (ja) 2022-08-25

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JP2019531379A Active JP7125940B2 (ja) 2016-12-19 2017-12-14 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2022075930A Withdrawn JP2022101695A (ja) 2016-12-19 2022-05-02 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2024095058A Active JP7728923B2 (ja) 2016-12-19 2024-06-12 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2025134907A Pending JP2025166161A (ja) 2016-12-19 2025-08-13 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

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JP2024095058A Active JP7728923B2 (ja) 2016-12-19 2024-06-12 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2025134907A Pending JP2025166161A (ja) 2016-12-19 2025-08-13 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

Country Status (31)

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US (2) US11382981B2 (enExample)
EP (3) EP4406601A3 (enExample)
JP (4) JP7125940B2 (enExample)
KR (2) KR102639633B1 (enExample)
CN (2) CN117298290A (enExample)
AU (1) AU2017382741B2 (enExample)
BR (1) BR112019012651A2 (enExample)
CA (1) CA3046801A1 (enExample)
CO (1) CO2019007398A2 (enExample)
DK (2) DK4122497T3 (enExample)
ES (2) ES2929498T3 (enExample)
FI (1) FI4122497T3 (enExample)
HR (2) HRP20240705T1 (enExample)
HU (2) HUE060065T2 (enExample)
IL (2) IL304284B2 (enExample)
LT (2) LT3554554T (enExample)
MA (1) MA65895B1 (enExample)
MD (2) MD3554554T2 (enExample)
MX (1) MX2019006989A (enExample)
MY (1) MY205600A (enExample)
NZ (1) NZ755422A (enExample)
PL (2) PL3554554T3 (enExample)
PT (2) PT3554554T (enExample)
RS (2) RS65583B1 (enExample)
SA (1) SA519402143B1 (enExample)
SG (1) SG10202100491QA (enExample)
SI (2) SI3554554T1 (enExample)
SM (2) SMT202200440T1 (enExample)
TW (1) TWI780095B (enExample)
WO (1) WO2018118627A1 (enExample)
ZA (1) ZA201903940B (enExample)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CN109563114B (zh) 2016-05-24 2022-08-12 萨勒普塔医疗公司 用于制备寡聚物的方法
SG10202101834QA (en) 2016-05-24 2021-04-29 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
HRP20240705T1 (hr) 2016-12-19 2024-08-30 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
AU2019316103B2 (en) 2018-08-02 2026-02-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
US20220152086A1 (en) * 2019-03-28 2022-05-19 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy with casimersen
JP2022528725A (ja) * 2019-04-18 2022-06-15 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを治療するための組成物
WO2020257489A1 (en) * 2019-06-19 2020-12-24 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
AU2020411964A1 (en) 2019-12-26 2022-06-16 National Center Of Neurology And Psychiatry Antisense nucleic acid that induces skipping of exon 50
PE20230237A1 (es) 2020-02-28 2023-02-07 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido que inducen la omision del exon 51
EP4168425A4 (en) * 2020-06-17 2024-06-19 Stealth BioTherapeutics Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT OF MUSCULAR DYSTROPHY
CA3211038A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
IL307937A (en) * 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
AU2022298028A1 (en) 2021-06-23 2023-12-21 National Center Of Neurology And Psychiatry Combination of antisense oligomers
WO2023282346A1 (ja) 2021-07-08 2023-01-12 日本新薬株式会社 析出抑制剤
IL310003A (en) 2021-07-08 2024-03-01 Nippon Shinyaku Co Ltd Nephrotoxicity reducing agent
JPWO2023282344A1 (enExample) 2021-07-08 2023-01-12
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN120129745A (zh) 2022-03-10 2025-06-10 日本新药株式会社 抗病毒反义低聚物
JP7523012B1 (ja) * 2023-05-17 2024-07-26 東亞合成株式会社 ペプチドフラグメント及びその利用
AU2024315761A1 (en) * 2023-08-03 2026-02-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Family Cites Families (108)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH445129A (fr) 1964-04-29 1967-10-15 Nestle Sa Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé
US3459731A (en) 1966-12-16 1969-08-05 Corn Products Co Cyclodextrin polyethers and their production
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic properties
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453259A (en) 1967-03-22 1969-07-01 Corn Products Co Cyclodextrin polyol ethers and their oxidation products
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
AU5661386A (en) 1985-03-15 1986-10-13 Stirchak, E. Stereoregular polynucleotide-binding polymers
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
KR0166088B1 (ko) 1990-01-23 1999-01-15 . 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
WO1994002595A1 (en) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
DE69435005T2 (de) 1993-05-11 2008-04-17 The University Of North Carolina At Chapel Hill Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
WO1999042091A2 (en) 1998-02-19 1999-08-26 Massachusetts Institute Of Technology Use of polycations as endosomolytic agents
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
JP2000125448A (ja) 1998-10-14 2000-04-28 Yazaki Corp 電気接続箱
JP2000256547A (ja) 1999-03-10 2000-09-19 Sumitomo Dow Ltd 耐熱性プラスチックカード用樹脂組成物
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
AU2281201A (en) 1999-12-29 2001-07-09 A. James Mixson Histidine copolymer and methods for using same
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
CA2459347C (en) 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
KR100464261B1 (ko) 2002-01-24 2005-01-03 주식회사 파나진 Pna 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
KR20030084444A (ko) 2002-04-26 2003-11-01 주식회사 파나진 Pna 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004044140A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceticals, Inc. 2’-substituted oligomeric compounds and compositions for use in gene modulations
EP2530155B1 (en) 2002-11-25 2016-03-16 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
SI2206781T1 (sl) 2004-06-28 2016-05-31 The University Of Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe
CA2605512A1 (en) 2005-04-22 2006-10-26 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary rna structure.
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US20070105807A1 (en) 2005-11-10 2007-05-10 Sazani Peter L Splice switch oligomers for TNF superfamily receptors and their use in treatment of disease
EP2019011A1 (en) 2006-05-17 2009-01-28 Svetlana Anatolevna Sokolova Transport means
AU2008271050B2 (en) 2007-06-29 2014-11-06 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2009008725A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
DK2203173T3 (en) 2007-10-26 2016-02-29 Academisch Ziekenhuis Leiden Materials and methods for prevention of muscle diseases
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
CA2884340C (en) 2007-11-15 2017-07-25 Sarepta Therapeutics, Inc. Method of synthesis of morpholino oligomers
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
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
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2762567B1 (en) * 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
CA2741629C (en) 2008-10-27 2022-07-05 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
CN102625840A (zh) 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
EP2479182B8 (en) 2009-09-16 2016-07-13 Wave Life Sciences Japan, Inc. Novel protecting group for synthesizing rna and derivative thereof
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス, インコーポレイテッド 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
EP2672977A1 (en) 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2012150960A1 (en) * 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
US9607308B2 (en) 2011-06-29 2017-03-28 American Express Travel Related Services Company, Inc. Spend based digital ad targeting and measurement
ES2535654T3 (es) 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
AU2012345638C1 (en) 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
CN108611349A (zh) 2011-12-28 2018-10-02 日本新药株式会社 反义核酸
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
DE102012101676A1 (de) 2012-02-29 2013-08-29 Klaus-Dieter Rösler Verfahren und Vorrichtung zum Bearbeiten von Formularen mit einer Datenverarbeitungsanlage
AU2013285698A1 (en) 2012-07-03 2015-02-19 Biomarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
US10233495B2 (en) 2012-09-27 2019-03-19 The Hospital For Sick Children Methods and compositions for screening and treating developmental disorders
KR20150099804A (ko) 2012-12-20 2015-09-01 사렙타 쎄러퓨틱스 인코퍼레이티드 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물
US9856474B2 (en) * 2013-01-16 2018-01-02 Iowa State University Research Foundation, Inc. Deep intronic target for splicing correction on spinal muscular atrophy gene
KR20230074606A (ko) 2013-03-14 2023-05-30 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
AU2014236140B2 (en) * 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
US20150329609A1 (en) 2014-03-03 2015-11-19 Sujata Acharjee Chimeric dystrophin proteins to treat dystrophinopathies
RU2702424C2 (ru) 2014-03-12 2019-10-08 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
WO2015194520A1 (ja) 2014-06-17 2015-12-23 日本新薬株式会社 アンチセンス核酸
JP6728156B2 (ja) 2014-11-02 2020-07-22 アークトゥラス・セラピューティクス・インコーポレイテッドArcturus Therapeutics,Inc. メッセンジャーuna分子およびその使用
CN108025089A (zh) 2015-07-22 2018-05-11 波涛生命科学有限公司 寡核苷酸组合物及其方法
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
SG10201912902TA (en) 2015-10-09 2020-02-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
NZ747685A (en) * 2016-04-29 2023-05-26 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
CN109477108A (zh) 2016-05-04 2019-03-15 波涛生命科学有限公司 寡核苷酸组合物和其方法
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
HRP20240705T1 (hr) * 2016-12-19 2024-08-30 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR102646318B1 (ko) 2016-12-19 2024-03-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии

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