CN107693797B - 肽寡核苷酸缀合物 - Google Patents

肽寡核苷酸缀合物 Download PDF

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Publication number
CN107693797B
CN107693797B CN201710707423.0A CN201710707423A CN107693797B CN 107693797 B CN107693797 B CN 107693797B CN 201710707423 A CN201710707423 A CN 201710707423A CN 107693797 B CN107693797 B CN 107693797B
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group
conjugate
carrier peptide
oligomer
sequence
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CN107693797A (zh
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贡纳·J·汉森
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Sarepta Therapeutics Inc
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Sarepta Therapeutics Inc
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Priority claimed from US13/107,528 external-priority patent/US9238042B2/en
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    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (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)
CN201710707423.0A 2011-05-05 2011-11-17 肽寡核苷酸缀合物 Active CN107693797B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US13/101,942 2011-05-05
US13/101,942 US20110269665A1 (en) 2009-06-26 2011-05-05 Compound and method for treating myotonic dystrophy
US13/107,528 2011-05-13
US13/107,528 US9238042B2 (en) 2010-05-13 2011-05-13 Antisense modulation of interleukins 17 and 23 signaling
CN201180071918.XA CN103619356B (zh) 2011-05-05 2011-11-17 肽寡核苷酸缀合物

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201180071918.XA Division CN103619356B (zh) 2011-05-05 2011-11-17 肽寡核苷酸缀合物

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CN107693797A CN107693797A (zh) 2018-02-16
CN107693797B true CN107693797B (zh) 2021-05-11

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CN201180071918.XA Active CN103619356B (zh) 2011-05-05 2011-11-17 肽寡核苷酸缀合物

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EP (1) EP2704749A1 (enExample)
JP (6) JP6478632B2 (enExample)
KR (3) KR102183273B1 (enExample)
CN (2) CN107693797B (enExample)
AU (6) AU2011367230B2 (enExample)
CA (2) CA3092114A1 (enExample)
IL (2) IL273838B (enExample)
WO (1) WO2012150960A1 (enExample)

Families Citing this family (90)

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Publication number Priority date Publication date Assignee Title
EP1766010B1 (en) 2004-06-28 2011-02-16 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
PT2049664E (pt) 2006-08-11 2012-01-03 Prosensa Technologies Bv Oligonucleotídeos de cadeia simples complementares dos elementos repetitivos para tratar perturbações genéticas associadas à instabilidade das repetições do adn
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
PL2203173T3 (pl) 2007-10-26 2016-06-30 Academisch Ziekenhuis Leiden Środki i sposoby przeciwdziałania zaburzeniom mięśni
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
KR20180118828A (ko) 2008-10-24 2018-10-31 사렙타 쎄러퓨틱스 인코퍼레이티드 Dmd를 위한 다중 엑손 스키핑 조성물
EP3431603A1 (en) 2009-11-12 2019-01-23 The University Of Western Australia Antisense molecules and methods for treating pathologies
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
JP2015500204A (ja) 2011-11-18 2015-01-05 サレプタ セラピューティクス， インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
AU2012345638C1 (en) 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
US10066228B2 (en) 2011-11-30 2018-09-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
PL2788487T3 (pl) 2011-12-08 2018-10-31 Sarepta Therapeutics, Inc. Analogi oligonukleotydów ukierunkowane na ludzki LMNA
JP2015509922A (ja) 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
EP3228712A1 (en) 2012-04-23 2017-10-11 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
EA201591178A1 (ru) 2012-12-20 2015-11-30 Сарепта Терапьютикс, Инк. Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии
EP2946013A1 (en) 2013-01-16 2015-11-25 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
CN110066793A (zh) 2013-03-14 2019-07-30 萨勒普塔医疗公司 用于治疗肌肉萎缩的外显子跳跃组合物
KR20230116945A (ko) 2013-03-14 2023-08-04 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
EP3015467B1 (en) * 2013-05-24 2024-12-04 Ajinomoto Co., Inc. Morpholino oligonucleotide manufacturing method
IL300444A (en) * 2013-09-05 2023-04-01 Sarepta Therapeutics Inc Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase
US10517869B2 (en) 2013-12-24 2019-12-31 Sentiss Pharma Private Limited Topical brimonidine tartrate ophthalmic solution
LT3118311T (lt) * 2014-03-12 2019-04-10 Nippon Shinyaku Co., Ltd. Priešprasminė nukleorūgštis
KR101661277B1 (ko) * 2014-03-17 2016-09-30 제주광어주식회사 한 개의 뉴크레오타이드 염기 변화를 통한 약독화 바이러스성 출혈패혈증 바이러스
CA2948373A1 (en) 2014-05-16 2015-11-19 Oregon State University Antisense antibacterial compounds and methods
EP3146047A4 (en) 2014-05-19 2018-04-04 Oregon State University Antisense antibacterial compounds and methods
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
US11293024B2 (en) 2014-12-31 2022-04-05 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
MX2017011004A (es) * 2015-02-27 2018-02-09 Sarepta Therapeutics Inc Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida.
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3297649B1 (en) 2015-05-19 2023-10-11 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
MA50829A (fr) 2015-06-01 2018-04-11 Sarepta Therapeutics Inc Exclusion d'exon induite pat technologie antisens dans le collagène de type vii
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
CN113461733A (zh) * 2015-08-05 2021-10-01 卫材R&D管理有限公司 用于制备均一低聚物的手性试剂
JP6987041B2 (ja) * 2015-08-28 2021-12-22 サレプタ セラピューティクス，インコーポレイテッド 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー
KR101841241B1 (ko) * 2015-09-10 2018-03-22 한양대학교 산학협력단 피부 질환에 대한 예방 또는 치료효과를 갖는 펩티드 융합체 및 이를 유효성분으로 하는 약학 조성물
WO2017062835A2 (en) 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
GB2545898B (en) * 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
CA3009342A1 (en) 2015-12-23 2017-06-29 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
US10907158B2 (en) 2015-12-23 2021-02-02 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
US11060089B2 (en) * 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
EP3449000A1 (en) 2016-04-29 2019-03-06 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human lmna
TW201811807A (zh) * 2016-06-30 2018-04-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
CN109152792B (zh) * 2016-06-30 2021-09-14 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
CN110337308B (zh) 2016-12-19 2023-09-01 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物
SMT202200403T1 (it) * 2016-12-19 2022-11-18 Sarepta Therapeutics Inc Coniugati di oligomeri per salto di esone per distrofia muscolare
MX2019006882A (es) * 2016-12-19 2019-08-16 Sarepta Therapeutics Inc Conjugados de oligomeros de omision de exon para distrofia muscular.
TW201919682A (zh) 2017-08-08 2019-06-01 西班牙商阿爾米雷爾有限公司 活化Ｎｒｆ２路徑的新穎化合物
AU2018337854B2 (en) * 2017-09-22 2025-01-16 Katarzyna JASTRZEBSKA Thiomorpholino oligonucleotides for the treatment of muscular dystrophy
EA201991450A1 (ru) * 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
WO2019067981A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
WO2019067975A1 (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
EP3697422A4 (en) * 2017-10-17 2021-08-11 Sarepta Therapeutics, Inc. CELL PENETRATION PEPTIDES FOR ANTISENS ADMINISTRATION
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
TW202002990A (zh) 2018-03-16 2020-01-16 美商薩羅塔治療公司 用於反義遞送之嵌合肽
WO2019209764A2 (en) * 2018-04-26 2019-10-31 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
EP4219717A3 (en) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
WO2019241385A2 (en) * 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy
TW202449155A (zh) 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
WO2020028254A1 (en) * 2018-07-30 2020-02-06 Sarepta Therapeutics, Inc. Trimeric peptides for antisense delivery
GB201812980D0 (en) * 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
WO2020115494A1 (en) * 2018-12-07 2020-06-11 Oxford University Innovation Limited Linkers
CA3122281A1 (en) 2018-12-13 2020-06-18 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
CN110724180B (zh) * 2019-10-17 2021-08-20 山东大学 一种抑制新生血管生成的多肽及其应用
MY209476A (en) 2019-12-26 2025-07-11 Nippon Shinyaku Co Ltd Antisense nucleic acid that induces skipping of exon 50
JPWO2021167107A1 (enExample) 2020-02-22 2021-08-26
CA3173034A1 (en) 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
EP4112083A4 (en) 2020-02-28 2024-06-26 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
WO2022061216A1 (en) * 2020-09-21 2022-03-24 Icahn School Of Medicine At Mount Sinai Archaea l30 proteins as universal influenza virus therapeutics
JP2024501800A (ja) 2020-12-23 2024-01-16 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エクソンスキッピングオリゴヌクレオチドコンジュゲートを含む組成物
AU2021427595A1 (en) * 2021-02-12 2023-09-28 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
EP4330395A1 (en) 2021-04-30 2024-03-06 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
MX2023014417A (es) 2021-06-23 2023-12-15 Nippon Shinyaku Co Ltd Combinacion de oligomeros antisentido.
AU2022306542A1 (en) 2021-07-08 2024-01-18 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
JPWO2023282345A1 (enExample) 2021-07-08 2023-01-12
TW202308664A (zh) 2021-07-08 2023-03-01 日商日本新藥股份有限公司 腎毒性減輕劑
US20240390508A1 (en) 2021-08-21 2024-11-28 Takeda Pharmaceutical Company Limited Human transferrin receptor binding peptide-drug conjugate
IL311025A (en) 2021-08-24 2024-04-01 Peptidream Inc Human transferrin receptor-binding antibody-peptide conjugate
IL311568A (en) 2021-09-30 2024-05-01 Sarepta Therapeutics Inc Antisense oligonucleotides having one or more abasic units
EP4419680A1 (en) 2021-10-22 2024-08-28 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
CA3252177A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. MORPHOLINO PHOSPHORODIAMIDATE OLIGOMER CONJUGATE
JP2025533454A (ja) 2022-09-21 2025-10-07 サレプタ セラピューティクス， インコーポレイテッド Ｄｍｄアンチセンスオリゴヌクレオチド介在性エクソンスキッピング効率
JP2025537104A (ja) 2022-11-02 2025-11-14 サレプタ セラピューティクス， インコーポレイテッド アンチセンスオリゴマーコンジュゲートの製剤化
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009005793A2 (en) * 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン，ジエ−ムス ポリヌクレオチド測定試薬と方法
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
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
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
WO1993001286A2 (en) 1991-06-28 1993-01-21 Massachusetts Institute Of Technology Localized oligonucleotide therapy
ATE173016T1 (de) 1992-08-21 1998-11-15 Biogen Inc Von tat abgeleitete transportpolypeptide
JPH0915828A (ja) 1995-07-03 1997-01-17 Fuji Photo Film Co Ltd 写真処理システム用ペーパーカッター
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
EP0975370B9 (en) 1997-05-21 2004-11-03 The Board Of Trustees Of The Leland Stanford Junior University Composition and method for enhancing transport across biological membranes
US6025140A (en) * 1997-07-24 2000-02-15 Perseptive Biosystems, Inc. Membrane-permeable constructs for transport across a lipid membrane
DE19933492B4 (de) * 1999-07-16 2008-01-10 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Konjugat zur Vermittlung eines zell-, kompartiment- oder membranspezifischen Transports von Wirksubstanzen, Verfahren zu dessen Herstellung und dessen Verwendung
US20020009491A1 (en) 2000-02-14 2002-01-24 Rothbard Jonathan B. Compositions and methods for enhancing drug delivery across biological membranes and tissues
CA2437983C (en) 2001-02-16 2011-10-25 Cellgate, Inc. Transporters comprising spaced arginine moieties
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
EP1629105B1 (en) 2003-04-29 2010-09-01 AVI BioPharma, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
AU2004276226B2 (en) 2003-08-05 2009-07-30 Avi Biopharma, Inc. Oligonucleotide analog and method for treating flavivirus infections
US20050171044A1 (en) 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
CA2565685A1 (en) * 2004-05-04 2005-12-15 Nastech Pharmaceutical Company Inc. Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US20050288246A1 (en) * 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
EP1766010B1 (en) 2004-06-28 2011-02-16 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US8129352B2 (en) 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US8357664B2 (en) 2004-10-26 2013-01-22 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
US7524829B2 (en) 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
WO2007002390A2 (en) 2005-06-23 2007-01-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
WO2007030691A2 (en) 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
EP1937278B1 (en) 2005-09-08 2012-07-25 AVI BioPharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US7582615B2 (en) 2006-03-07 2009-09-01 Avi Biopharma, Inc. Antisense antiviral compound and method for treating arenavirus infection
HUE035799T2 (en) 2006-05-10 2018-05-28 Sarepta Therapeutics Inc Cationic oligonucleotide analogues containing subunits
PT2049664E (pt) 2006-08-11 2012-01-03 Prosensa Technologies Bv Oligonucleotídeos de cadeia simples complementares dos elementos repetitivos para tratar perturbações genéticas associadas à instabilidade das repetições do adn
CA2981308C (en) 2006-09-21 2020-12-22 University Of Rochester Compositions and methods related to protein displacement therapy for myotonic dystrophy
JP5227803B2 (ja) 2006-11-24 2013-07-03 ハイクス ラボラトリーズ合同会社 スピロキノン化合物及び医薬組成物
WO2009005783A1 (en) * 2007-06-28 2009-01-08 Blanchette Rockefeller Neurosciences Institute Peptides, compositions and methods for reducing beta-amyloid-mediated apoptosis
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
MX2010004955A (es) 2007-11-15 2010-06-30 Avi Biopharma Inc Metodo de sintesis de oligomeros de morfolina.
AU2008340943A1 (en) * 2007-12-20 2009-07-02 Angiochem Inc. Polypeptide-nucleic acid conjugates and uses thereof
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
KR20180118828A (ko) 2008-10-24 2018-10-31 사렙타 쎄러퓨틱스 인코퍼레이티드 Dmd를 위한 다중 엑손 스키핑 조성물
WO2010072228A1 (en) * 2008-12-22 2010-07-01 Xigen S.A. Novel transporter constructs and transporter cargo conjugate molecules
WO2010120820A1 (en) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
KR20200047790A (ko) 2009-06-17 2020-05-07 바이오젠 엠에이 인코포레이티드 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
WO2011143608A1 (en) * 2010-05-13 2011-11-17 Avi Biopharma, Inc. Antisense modulation of interleukins 17 and 23 signaling

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009005793A2 (en) * 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
"Construction of a novel chimera consisting of a chelator-containing Tat peptide conjugated to a morpholino antisense oligomer for technetium-99m labeling and accelerating cellular kinetics";Yu-Min Zhang et al.;《Nuclear Medicine and Biology》;20060228;第33卷(第2期);第263-269页 *

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