CN104024414A - 反义核酸 - Google Patents

反义核酸 Download PDF

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Publication number
CN104024414A
CN104024414A CN201280064590.3A CN201280064590A CN104024414A CN 104024414 A CN104024414 A CN 104024414A CN 201280064590 A CN201280064590 A CN 201280064590A CN 104024414 A CN104024414 A CN 104024414A
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CN
China
Prior art keywords
exon
forms
sequence
dystrophin gene
nucleotide
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.)
Withdrawn
Application number
CN201280064590.3A
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English (en)
Chinese (zh)
Inventor
渡边直树
濑尾春奈
武田伸一
永田哲也
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
Original Assignee
Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Nippon Shinyaku Co Ltd, National Center of Neurology and Psychiatry filed Critical Nippon Shinyaku Co Ltd
Priority to CN201910167665.4A priority Critical patent/CN110055244A/zh
Priority to CN201711192063.1A priority patent/CN107881175B/zh
Priority to CN202410462539.2A priority patent/CN118207212A/zh
Priority to CN201810389811.3A priority patent/CN108486116A/zh
Priority to CN201810389950.6A priority patent/CN108611349A/zh
Priority to CN201810389742.6A priority patent/CN108588073A/zh
Priority to CN202311738073.6A priority patent/CN117721110A/zh
Priority to CN201910167486.0A priority patent/CN110055243B/zh
Priority to CN201910167466.3A priority patent/CN110079525A/zh
Publication of CN104024414A publication Critical patent/CN104024414A/zh
Withdrawn legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CN201280064590.3A 2011-12-28 2012-12-27 反义核酸 Withdrawn CN104024414A (zh)

Priority Applications (9)

Application Number Priority Date Filing Date Title
CN201910167665.4A CN110055244A (zh) 2011-12-28 2012-12-27 反义核酸
CN201711192063.1A CN107881175B (zh) 2011-12-28 2012-12-27 反义核酸
CN202410462539.2A CN118207212A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389811.3A CN108486116A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389950.6A CN108611349A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389742.6A CN108588073A (zh) 2011-12-28 2012-12-27 反义核酸
CN202311738073.6A CN117721110A (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167486.0A CN110055243B (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167466.3A CN110079525A (zh) 2011-12-28 2012-12-27 反义核酸

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JP2011-288040 2011-12-28
JP2011288040 2011-12-28
JP2012043092 2012-02-29
JP2012-043092 2012-02-29
PCT/JP2012/084295 WO2013100190A1 (ja) 2011-12-28 2012-12-27 アンチセンス核酸

Related Child Applications (9)

Application Number Title Priority Date Filing Date
CN201810389742.6A Division CN108588073A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389811.3A Division CN108486116A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389950.6A Division CN108611349A (zh) 2011-12-28 2012-12-27 反义核酸
CN201711192063.1A Division CN107881175B (zh) 2011-12-28 2012-12-27 反义核酸
CN202410462539.2A Division CN118207212A (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167466.3A Division CN110079525A (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167665.4A Division CN110055244A (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167486.0A Division CN110055243B (zh) 2011-12-28 2012-12-27 反义核酸
CN202311738073.6A Division CN117721110A (zh) 2011-12-28 2012-12-27 反义核酸

Publications (1)

Publication Number Publication Date
CN104024414A true CN104024414A (zh) 2014-09-03

Family

ID=48697653

Family Applications (10)

Application Number Title Priority Date Filing Date
CN201810389950.6A Pending CN108611349A (zh) 2011-12-28 2012-12-27 反义核酸
CN202410462539.2A Pending CN118207212A (zh) 2011-12-28 2012-12-27 反义核酸
CN201711192063.1A Active CN107881175B (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167466.3A Pending CN110079525A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389742.6A Pending CN108588073A (zh) 2011-12-28 2012-12-27 反义核酸
CN201280064590.3A Withdrawn CN104024414A (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167486.0A Active CN110055243B (zh) 2011-12-28 2012-12-27 反义核酸
CN202311738073.6A Pending CN117721110A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389811.3A Pending CN108486116A (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167665.4A Withdrawn CN110055244A (zh) 2011-12-28 2012-12-27 反义核酸

Family Applications Before (5)

Application Number Title Priority Date Filing Date
CN201810389950.6A Pending CN108611349A (zh) 2011-12-28 2012-12-27 反义核酸
CN202410462539.2A Pending CN118207212A (zh) 2011-12-28 2012-12-27 反义核酸
CN201711192063.1A Active CN107881175B (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167466.3A Pending CN110079525A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389742.6A Pending CN108588073A (zh) 2011-12-28 2012-12-27 反义核酸

Family Applications After (4)

Application Number Title Priority Date Filing Date
CN201910167486.0A Active CN110055243B (zh) 2011-12-28 2012-12-27 反义核酸
CN202311738073.6A Pending CN117721110A (zh) 2011-12-28 2012-12-27 反义核酸
CN201810389811.3A Pending CN108486116A (zh) 2011-12-28 2012-12-27 反义核酸
CN201910167665.4A Withdrawn CN110055244A (zh) 2011-12-28 2012-12-27 反义核酸

Country Status (12)

Country Link
US (5) US9512424B2 (enExample)
EP (2) EP2799548B1 (enExample)
JP (6) JP6141770B2 (enExample)
KR (2) KR102240139B1 (enExample)
CN (10) CN108611349A (enExample)
AU (2) AU2012360702C1 (enExample)
CA (2) CA2861247C (enExample)
ES (1) ES2748868T3 (enExample)
IN (1) IN2014DN06220A (enExample)
RU (3) RU2619184C2 (enExample)
TW (1) TWI606059B (enExample)
WO (1) WO2013100190A1 (enExample)

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106459955A (zh) * 2014-03-12 2017-02-22 日本新药株式会社 反义核酸
CN108026531A (zh) * 2015-09-15 2018-05-11 日本新药株式会社 反义核酸
CN109311920A (zh) * 2016-05-24 2019-02-05 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
CN109311919A (zh) * 2016-06-30 2019-02-05 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体
CN109563114A (zh) * 2016-05-24 2019-04-02 萨勒普塔医疗公司 用于制备寡聚物的方法
CN114901823A (zh) * 2019-12-26 2022-08-12 日本新药株式会社 诱导外显子50的跳读的反义核酸

Families Citing this family (49)

* Cited by examiner, † Cited by third party
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PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
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
SMT201900159T1 (it) 2008-10-24 2019-05-10 Sarepta Therapeutics Inc Composizioni di salto di esone per dmd
KR20230137491A (ko) 2009-11-12 2023-10-04 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CN108611349A (zh) * 2011-12-28 2018-10-02 日本新药株式会社 反义核酸
BR112014018427B1 (pt) 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
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US10174319B2 (en) * 2014-05-19 2019-01-08 Knc Laboratories Co., Ltd. Nucleic acid drug for inducing skipping of variant exon of CD44 gene and increasing expression of normal type CD44 mRNA
CN111575282B (zh) * 2014-06-17 2024-05-14 日本新药株式会社 反义核酸
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MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3359668A4 (en) 2015-10-09 2019-06-05 Sarepta Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF
NZ781029A (en) 2015-10-09 2025-07-25 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
WO2018107003A1 (en) * 2016-12-08 2018-06-14 The Board Of Regents Of The University Of Texas System Dmd reporter models containing humanized duschene muscular dystrophy mutations
SG10202012839TA (en) 2016-12-19 2021-01-28 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
MD3554554T2 (ro) 2016-12-19 2022-12-31 Sarepta Therapeutics Inc Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară
BR112019012664A2 (pt) 2016-12-19 2020-01-21 Sarepta Therapeutics Inc conjugados de oligômero de salto de éxon para distrofia muscular
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
EP3687577A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
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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
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
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
IL318474A (en) * 2018-12-13 2025-03-01 Sarepta Therapeutics Inc Axon-skipping oligomer complexes for muscular dystrophy
JP7185239B2 (ja) * 2018-12-25 2022-12-07 国立研究開発法人国立精神・神経医療研究センター 随時尿中細胞を用いた筋系細胞の誘導方法
EP3959319A4 (en) 2019-04-25 2023-06-07 Avidity Biosciences, Inc. NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING
CN115151642A (zh) 2019-12-19 2022-10-04 日本新药株式会社 能够进行外显子跳读的反义核酸
KR20240004609A (ko) 2021-04-30 2024-01-11 사렙타 쎄러퓨틱스 인코퍼레이티드 근이영양증에 대한 치료 방법
WO2022239863A1 (ja) 2021-05-13 2022-11-17 国立大学法人千葉大学 アンチセンスオリゴマー
MX2023014417A (es) 2021-06-23 2023-12-15 Nippon Shinyaku Co Ltd Combinacion de oligomeros antisentido.
KR20240035502A (ko) 2021-07-08 2024-03-15 니뽄 신야쿠 가부시키가이샤 신독성 경감제
EP4368176A4 (en) 2021-07-08 2025-06-25 Nippon Shinyaku Co., Ltd. Nephrotoxicity reducing agent
JPWO2023282346A1 (enExample) 2021-07-08 2023-01-12
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
MX2024000494A (es) * 2021-07-09 2024-04-10 Dyne Therapeutics Inc Complejos dirigidos al tratamiento de musculos, y usos de los mismos para tratar distrofinopatías.
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