WO2012150960A1 - Peptide oligonucleotide conjugates - Google Patents

Peptide oligonucleotide conjugates Download PDF

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Publication number
WO2012150960A1
WO2012150960A1 PCT/US2011/061282 US2011061282W WO2012150960A1 WO 2012150960 A1 WO2012150960 A1 WO 2012150960A1 US 2011061282 W US2011061282 W US 2011061282W WO 2012150960 A1 WO2012150960 A1 WO 2012150960A1
Authority
WO
WIPO (PCT)
Prior art keywords
conjugate
occurrence
alkyl
independently
carrier peptide
Prior art date
Application number
PCT/US2011/061282
Other languages
English (en)
French (fr)
Inventor
Gunnar J. Hanson
Original Assignee
Avi Biopharma, Inc.
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
Priority claimed from US13/101,942 external-priority patent/US20110269665A1/en
Priority claimed from US13/107,528 external-priority patent/US9238042B2/en
Priority to JP2014509281A priority Critical patent/JP6478632B2/ja
Priority to AU2011367230A priority patent/AU2011367230B2/en
Priority to CN201180071918.XA priority patent/CN103619356B/zh
Priority to EP11793913.2A priority patent/EP2704749A1/en
Priority to IL273838A priority patent/IL273838B/en
Priority to KR1020197019623A priority patent/KR102229650B1/ko
Application filed by Avi Biopharma, Inc. filed Critical Avi Biopharma, Inc.
Priority to CA2834128A priority patent/CA2834128A1/en
Priority to KR1020137032241A priority patent/KR102183273B1/ko
Priority to KR1020217007616A priority patent/KR102339196B1/ko
Publication of WO2012150960A1 publication Critical patent/WO2012150960A1/en
Priority to IL229227A priority patent/IL229227B/en
Priority to AU2017206179A priority patent/AU2017206179A1/en
Priority to AU2019204913A priority patent/AU2019204913A1/en
Priority to AU2021202224A priority patent/AU2021202224A1/en
Priority to AU2023203112A priority patent/AU2023203112A1/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biochemistry (AREA)
  • Pulmonology (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Urology & Nephrology (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PCT/US2011/061282 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates WO2012150960A1 (en)

Priority Applications (14)

Application Number Priority Date Filing Date Title
KR1020137032241A KR102183273B1 (ko) 2011-05-05 2011-11-17 펩타이드 올리고뉴클레오타이드 접합체
KR1020217007616A KR102339196B1 (ko) 2011-05-05 2011-11-17 펩타이드 올리고뉴클레오타이드 접합체
CA2834128A CA2834128A1 (en) 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates
CN201180071918.XA CN103619356B (zh) 2011-05-05 2011-11-17 肽寡核苷酸缀合物
EP11793913.2A EP2704749A1 (en) 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates
IL273838A IL273838B (en) 2011-05-05 2011-11-17 Peptide and oligonucleotide conjugates, preparations containing them and their uses
KR1020197019623A KR102229650B1 (ko) 2011-05-05 2011-11-17 펩타이드 올리고뉴클레오타이드 접합체
JP2014509281A JP6478632B2 (ja) 2011-05-05 2011-11-17 ペプチドオリゴヌクレオチドコンジュゲート
AU2011367230A AU2011367230B2 (en) 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates
IL229227A IL229227B (en) 2011-05-05 2013-11-04 Peptide and oligonucleotide conjugates, preparations containing them and their uses
AU2017206179A AU2017206179A1 (en) 2011-05-05 2017-07-18 Peptide oligonucleotide conjugates
AU2019204913A AU2019204913A1 (en) 2011-05-05 2019-07-09 Peptide oligonucleotide conjugates
AU2021202224A AU2021202224A1 (en) 2011-05-05 2021-04-13 Peptide oligonucleotide conjugates
AU2023203112A AU2023203112A1 (en) 2011-05-05 2023-05-18 Peptide oligonucleotide conjugates

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US13/101,942 US20110269665A1 (en) 2009-06-26 2011-05-05 Compound and method for treating myotonic dystrophy
US13/101,942 2011-05-05
US13/107,528 US9238042B2 (en) 2010-05-13 2011-05-13 Antisense modulation of interleukins 17 and 23 signaling
US13/107,528 2011-05-13

Publications (1)

Publication Number Publication Date
WO2012150960A1 true WO2012150960A1 (en) 2012-11-08

Family

ID=45218873

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/061282 WO2012150960A1 (en) 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates

Country Status (8)

Country Link
EP (1) EP2704749A1 (ko)
JP (6) JP6478632B2 (ko)
KR (3) KR102183273B1 (ko)
CN (2) CN103619356B (ko)
AU (5) AU2011367230B2 (ko)
CA (2) CA3092114A1 (ko)
IL (2) IL273838B (ko)
WO (1) WO2012150960A1 (ko)

Cited By (72)

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WO2013082551A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
WO2013082548A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
WO2014100714A1 (en) 2012-12-20 2014-06-26 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
WO2014113540A1 (en) 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
CN104039739A (zh) * 2011-11-18 2014-09-10 萨勒普塔医疗公司 功能改性的寡核苷酸及其亚单元
WO2014144978A2 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
WO2014153220A2 (en) 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
WO2014153240A2 (en) 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
WO2015035231A1 (en) 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
US9018368B2 (en) 2004-06-28 2015-04-28 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US9066967B2 (en) 2011-12-08 2015-06-30 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human LMNA
WO2015137409A1 (ja) * 2014-03-12 2015-09-17 日本新薬株式会社 アンチセンス核酸
US9228187B2 (en) 2009-11-12 2016-01-05 The University Of Western Australia Antisense molecules and methods for treating pathologies
CN105228999A (zh) * 2013-05-24 2016-01-06 味之素株式会社 吗啉代寡核苷酸的制备方法
US9234198B1 (en) 2008-10-24 2016-01-12 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
US9499818B2 (en) 2007-10-26 2016-11-22 BioMarin Technologies, B.V. Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene
WO2016187425A1 (en) 2015-05-19 2016-11-24 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2016196670A1 (en) 2015-06-01 2016-12-08 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type vii collagen
WO2017112888A1 (en) 2015-12-23 2017-06-29 David Greenberg Antisense antibacterial compounds and methods
US9745576B2 (en) 2012-04-23 2017-08-29 Biomarin Technologies B.V. RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
US9790495B2 (en) 2014-05-16 2017-10-17 Oregon State University Antisense antibacterial compounds and methods
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
WO2017190041A1 (en) 2016-04-29 2017-11-02 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human lmna
US9890379B2 (en) 2006-08-11 2018-02-13 Biomarin Technologies B.V. Treatment of genetic disorders associated with DNA repeat instability
WO2018118662A1 (en) * 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2018118627A1 (en) * 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2018118599A1 (en) * 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3271372A4 (en) * 2015-03-18 2018-12-19 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in myostatin
US10179912B2 (en) 2012-01-27 2019-01-15 Biomarin Technologies B.V. RNA modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
US20190015440A1 (en) * 2015-08-28 2019-01-17 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
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WO2019030298A1 (en) 2017-08-08 2019-02-14 Almirall, S.A. NEW COMPOUNDS THAT ACTIVATE THE NRF2 PATH
WO2019059973A1 (en) * 2017-09-22 2019-03-28 Sarepta Therapeutics, Inc. OLIGOMERIC CONJUGATES FOR THE EXON JUMP FOR MUSCLE DYSTROPHY
US10246707B2 (en) 2008-05-14 2019-04-02 Biomarin Technologies B.V. Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
WO2019067981A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
WO2019067979A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
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US10385092B2 (en) 2010-09-01 2019-08-20 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
US10391098B2 (en) 2014-05-19 2019-08-27 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
US10421969B2 (en) 2011-10-04 2019-09-24 Royal Holloway And Bedford New College Oligomers
US10517869B2 (en) 2013-12-24 2019-12-31 Sentiss Pharma Private Limited Topical brimonidine tartrate ophthalmic solution
WO2020023688A1 (en) 2018-07-27 2020-01-30 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
US10626396B2 (en) 2005-02-09 2020-04-21 Sarepta Therapeutics, Inc. Antisense composition and method for treating muscle atrophy
WO2020123574A1 (en) 2018-12-13 2020-06-18 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2020138509A1 (en) 2018-12-28 2020-07-02 Nippon Shinyaku Co., Ltd. Myostatin signal inhibitor
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US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
EP3684933A4 (en) * 2017-09-22 2021-06-23 The Regents of the University of Colorado, A Body Corporate OLIGONUCLEOTIDES THIOMORPHOLINO FOR THE TREATMENT OF MUSCLE DYSTROPHY
WO2021132591A1 (ja) 2019-12-26 2021-07-01 日本新薬株式会社 エクソン５０のスキッピングを誘導するアンチセンス核酸
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WO2022270585A1 (ja) 2021-06-23 2022-12-29 日本新薬株式会社 アンチセンスオリゴマーの組み合わせ
WO2023282344A1 (ja) 2021-07-08 2023-01-12 日本新薬株式会社 腎毒性軽減剤
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WO2023055774A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
WO2023070086A1 (en) 2021-10-22 2023-04-27 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
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US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
KR101661277B1 (ko) * 2014-03-17 2016-09-30 제주광어주식회사 한 개의 뉴크레오타이드 염기 변화를 통한 약독화 바이러스성 출혈패혈증 바이러스
US20180216111A1 (en) * 2015-02-27 2018-08-02 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
JP6978406B2 (ja) * 2015-08-05 2021-12-08 エーザイ・アール・アンド・ディー・マネジメント株式会社 均質オリゴマーを調製するためのキラル試薬
WO2017205879A2 (en) * 2016-05-24 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
SG11201810143PA (en) * 2016-06-30 2019-01-30 Sarepta Therapeutics Inc Exon skipping oligomers for muscular dystrophy
JP7320500B2 (ja) * 2017-10-17 2023-08-03 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達のための細胞透過性ペプチド
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