AU2011367230B2 - Peptide oligonucleotide conjugates - Google Patents

Peptide oligonucleotide conjugates Download PDF

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Publication number
AU2011367230B2
AU2011367230B2 AU2011367230A AU2011367230A AU2011367230B2 AU 2011367230 B2 AU2011367230 B2 AU 2011367230B2 AU 2011367230 A AU2011367230 A AU 2011367230A AU 2011367230 A AU2011367230 A AU 2011367230A AU 2011367230 B2 AU2011367230 B2 AU 2011367230B2
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AU
Australia
Prior art keywords
conjugate
occurrence
alkyl
carrier peptide
independently
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AU2011367230A
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AU2011367230A1 (en
Inventor
Gunnar J. Hanson
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Sarepta Therapeutics Inc
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Sarepta Therapeutics Inc
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Priority claimed from US13/101,942 external-priority patent/US20110269665A1/en
Priority claimed from US13/107,528 external-priority patent/US9238042B2/en
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of AU2011367230A1 publication Critical patent/AU2011367230A1/en
Priority to AU2017206179A priority Critical patent/AU2017206179A1/en
Application granted granted Critical
Publication of AU2011367230B2 publication Critical patent/AU2011367230B2/en
Priority to AU2019204913A priority patent/AU2019204913A1/en
Priority to AU2021202224A priority patent/AU2021202224A1/en
Priority to AU2023203112A priority patent/AU2023203112B2/en
Priority to AU2025226744A priority patent/AU2025226744A1/en
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/16Antivirals 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (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)
  • Rheumatology (AREA)
  • Urology & Nephrology (AREA)
  • Pain & Pain Management (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)
AU2011367230A 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates Active AU2011367230B2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
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 AU2023203112B2 (en) 2011-05-05 2023-05-18 Peptide oligonucleotide conjugates
AU2025226744A AU2025226744A1 (en) 2011-05-05 2025-09-04 Peptide oligonucleotide conjugates

Applications Claiming Priority (5)

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 2011-05-13
US13/107,528 US9238042B2 (en) 2010-05-13 2011-05-13 Antisense modulation of interleukins 17 and 23 signaling
PCT/US2011/061282 WO2012150960A1 (en) 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2017206179A Division AU2017206179A1 (en) 2011-05-05 2017-07-18 Peptide oligonucleotide conjugates

Publications (2)

Publication Number Publication Date
AU2011367230A1 AU2011367230A1 (en) 2013-12-05
AU2011367230B2 true AU2011367230B2 (en) 2017-08-10

Family

ID=45218873

Family Applications (6)

Application Number Title Priority Date Filing Date
AU2011367230A Active AU2011367230B2 (en) 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates
AU2017206179A Abandoned AU2017206179A1 (en) 2011-05-05 2017-07-18 Peptide oligonucleotide conjugates
AU2019204913A Abandoned AU2019204913A1 (en) 2011-05-05 2019-07-09 Peptide oligonucleotide conjugates
AU2021202224A Abandoned AU2021202224A1 (en) 2011-05-05 2021-04-13 Peptide oligonucleotide conjugates
AU2023203112A Active AU2023203112B2 (en) 2011-05-05 2023-05-18 Peptide oligonucleotide conjugates
AU2025226744A Pending AU2025226744A1 (en) 2011-05-05 2025-09-04 Peptide oligonucleotide conjugates

Family Applications After (5)

Application Number Title Priority Date Filing Date
AU2017206179A Abandoned AU2017206179A1 (en) 2011-05-05 2017-07-18 Peptide oligonucleotide conjugates
AU2019204913A Abandoned AU2019204913A1 (en) 2011-05-05 2019-07-09 Peptide oligonucleotide conjugates
AU2021202224A Abandoned AU2021202224A1 (en) 2011-05-05 2021-04-13 Peptide oligonucleotide conjugates
AU2023203112A Active AU2023203112B2 (en) 2011-05-05 2023-05-18 Peptide oligonucleotide conjugates
AU2025226744A Pending AU2025226744A1 (en) 2011-05-05 2025-09-04 Peptide oligonucleotide conjugates

Country Status (8)

Country Link
EP (1) EP2704749A1 (enExample)
JP (6) JP6478632B2 (enExample)
KR (3) KR102229650B1 (enExample)
CN (2) CN103619356B (enExample)
AU (6) AU2011367230B2 (enExample)
CA (2) CA3092114A1 (enExample)
IL (2) IL273838B (enExample)
WO (1) WO2012150960A1 (enExample)

Families Citing this family (90)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
USRE47769E1 (en) 2004-06-28 2019-12-17 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
ES2373246T3 (es) 2006-08-11 2012-02-01 Prosensa Technologies B.V. Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn.
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling 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
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
WO2011057350A1 (en) 2009-11-12 2011-05-19 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
EA035030B1 (ru) * 2011-11-18 2020-04-20 Сарепта Терапьютикс, Инк. Модифицированные морфолиновые аналоги олигонуклеотидов
JP6317675B2 (ja) 2011-11-30 2018-04-25 サレプタ セラピューティクス, インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
ES2727481T3 (es) 2011-11-30 2019-10-16 Sarepta Therapeutics Inc Inclusión inducida de exón en atrofia muscular espinal
WO2013086444A2 (en) 2011-12-08 2013-06-13 Sarepta Therapeutics, Inc. Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human lmna
CN111893117B (zh) 2012-01-27 2024-06-04 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
HRP20171254T1 (hr) 2012-04-23 2018-01-12 Biomarin Technologies B.V. Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja
BR112015014987A2 (pt) 2012-12-20 2017-08-15 Sarepta Therapeutics Inc Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular
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
SMT201900139T1 (it) 2013-03-14 2019-05-10 Sarepta Therapeutics Inc Composizioni di salto dell'esone per il trattamento della distrofia muscolare
US9217148B2 (en) * 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
MX373959B (es) 2013-03-15 2020-07-13 Sarepta Therapeutics Inc Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).
EP3015467B1 (en) * 2013-05-24 2024-12-04 Ajinomoto Co., Inc. Morpholino oligonucleotide manufacturing method
MX384377B (es) * 2013-09-05 2025-03-14 Sarepta Therapeutics Inc Inclusión del exón 2 inducida por antisentido en la alfa-glucosidasa ácida.
UA117768C2 (uk) 2013-12-24 2018-09-25 Сентісс Фарма Прайвет Лімітед Офтальмологічний розчин тартрату бримонідину для місцевого застосування
EP3514234A1 (en) * 2014-03-12 2019-07-24 Nippon Shinyaku Co., Ltd. Antisense nucleic acid
KR101661277B1 (ko) * 2014-03-17 2016-09-30 제주광어주식회사 한 개의 뉴크레오타이드 염기 변화를 통한 약독화 바이러스성 출혈패혈증 바이러스
WO2015175977A2 (en) 2014-05-16 2015-11-19 Geller Bruce L Antisense antibacterial compounds and methods
US10391098B2 (en) 2014-05-19 2019-08-27 Board Of Regents, The University Of Texas System 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
EP3240794A4 (en) 2014-12-31 2018-10-31 Oregon State University Antisense antibacterial compounds and methods
MA41759A (fr) * 2015-02-27 2018-01-03 Univ Murdoch Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
MX388743B (es) * 2015-05-19 2025-03-20 Sarepta Therapeutics Inc Conjugados oligonucleótido-péptido.
HK1253335A1 (zh) 2015-06-01 2019-06-14 Sarepta Therapeutics, Inc. 在vii型胶原蛋白中反义诱导外显子的排除
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
MA44209B1 (fr) * 2015-08-05 2022-03-31 Eisai R&D Man Co Ltd Procede de preparation d'un oligomere phosphorodiamidate substantiellement diastereomeriquement pure, un oligomere phosphorodiamidate faite par un tel procede et une composition pharmaceutique comprenant un tel oligomere phosphorodiamidate
WO2017040271A1 (en) * 2015-08-28 2017-03-09 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
EP3336098B1 (en) * 2015-09-10 2021-08-11 IUCF-HYU (Industry-University Cooperation Foundation Hanyang University) Skin permeable peptide and method for using same
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
GB2545898B (en) * 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
JP7393121B2 (ja) 2015-12-23 2023-12-06 オレゴン ステイト ユニバーシティー アンチセンス抗菌化合物および方法
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
MA45616A (fr) 2016-04-29 2019-03-06 Sarepta Therapeutics Inc Analogues d'oligonucléotide ciblant lmna humain
AU2017270598B2 (en) * 2016-05-24 2022-12-01 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
JP2019525742A (ja) * 2016-06-30 2019-09-12 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマー
EP3554553B1 (en) * 2016-12-19 2022-07-20 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
CA3047010A1 (en) * 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR20240006023A (ko) * 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
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
EA201991450A1 (ru) * 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
JP7441455B2 (ja) * 2017-09-22 2024-03-01 ザ リージェンツ オブ ザ ユニバーシティ オブ コロラド,ア ボディー コーポレイト 筋ジストロフィーの処置のためのチオモルホリノオリゴヌクレオチド
US20210145852A1 (en) 2017-09-28 2021-05-20 Sarepta Therapeutics, Inc. Combination Therapies for Treating Muscular Dystrophy
JP2020536057A (ja) 2017-09-28 2020-12-10 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処置するための併用療法
US20200248178A1 (en) 2017-09-28 2020-08-06 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
MA50834A (fr) * 2017-10-17 2020-08-26 Massachusetts Inst Technology Peptides de pénétration cellulaire pour administration antisens
JP7394753B2 (ja) 2017-10-18 2023-12-08 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化合物
EP3765030A4 (en) 2018-03-16 2022-01-26 Sarepta Therapeutics, Inc. CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION
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
TW202020153A (zh) 2018-07-27 2020-06-01 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
TW202019442A (zh) 2018-07-30 2020-06-01 美商薩羅塔治療公司 用於反義遞送之三聚合肽
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
EP3890783A1 (en) 2018-12-07 2021-10-13 Oxford University Innovation Limited Linkers
EA202191601A1 (ru) 2018-12-13 2022-01-19 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
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
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
CN118109469A (zh) 2019-12-26 2024-05-31 日本新药株式会社 诱导外显子50的跳读的反义核酸
US20230203098A1 (en) 2020-02-22 2023-06-29 Jcr Pharmaceuticals Co., Ltd. Human transferrin receptor binding peptide
BR112022017066A2 (pt) 2020-02-28 2022-11-16 Nippon Shinyaku Co Ltd Ácido nucleico antissentido indutor de salto do éxon 51
BR112022016238A2 (pt) 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular smn2
US20240024414A1 (en) * 2020-09-21 2024-01-25 Icahn School Of Medicine At Mount Sinai Archaea l30 proteins as universal influenza virus therapeutics
US20240327831A1 (en) 2020-12-23 2024-10-03 Sarepta Therapeutics, Inc. Compositions comprising exon skipping oligonucleotide conjugates for treating 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
TW202307208A (zh) 2021-06-23 2023-02-16 日商日本新藥股份有限公司 反義寡聚物之組合
CN118201606A (zh) 2021-07-08 2024-06-14 日本新药株式会社 肾毒性减轻剂
TW202317147A (zh) 2021-07-08 2023-05-01 日商日本新藥股份有限公司 析出抑制劑
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
WO2023026994A1 (ja) 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド-薬物コンジュゲート
CA3229962A1 (en) 2021-08-24 2023-03-02 Peptidream Inc. Human transferrin receptor-binding antibody-peptide conjugate
US20240425864A1 (en) 2021-09-30 2024-12-26 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
JP2024539223A (ja) 2021-10-22 2024-10-28 サレプタ セラピューティクス, インコーポレイテッド 末梢ミエリンタンパク質22関連疾患の処置のためのモルフォリノオリゴマー
US20250195667A1 (en) 2022-03-17 2025-06-19 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
WO2024097822A1 (en) 2022-11-02 2024-05-10 Sarepta Therapeutics, Inc. Formulation of an antisense oligomer conjugate
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies

Citations (4)

* 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
WO2009144481A2 (en) * 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2011143608A1 (en) * 2010-05-13 2011-11-17 Avi Biopharma, Inc. Antisense modulation of interleukins 17 and 23 signaling

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. Polynucleotide assay reagent and method
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
AU659482B2 (en) 1991-06-28 1995-05-18 Massachusetts Institute Of Technology Localized oligonucleotide therapy
EP0903408A3 (en) 1992-08-21 2005-11-02 Biogen, Inc. Tat-derived transport polypeptide
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
ES2210761T3 (es) 1997-05-21 2004-07-01 The Board Of Trustees Of The Leland Stanford Junior University Composicion y procedimiento para mejorar el transporte a traves de las membranas biologicas.
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
WO2002065986A2 (en) 2001-02-16 2002-08-29 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
ES2351976T3 (es) 2003-04-29 2011-02-14 Avi Biopharma, Inc. Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células.
ATE491791T1 (de) 2003-08-05 2011-01-15 Avi Biopharma Inc Oligonukleotidanalog und verfahren zur behandlung von flavivirusinfektionen
US20050171044A1 (en) 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
EP1750775A2 (en) * 2004-05-04 2007-02-14 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
USRE47769E1 (en) 2004-06-28 2019-12-17 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
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
DK1910395T3 (da) 2005-06-23 2013-02-04 Isis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning
US8329668B2 (en) 2005-09-08 2012-12-11 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8524676B2 (en) 2005-09-08 2013-09-03 Sarepta Therapeutics, Inc. Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
CA2644262C (en) 2006-03-07 2016-01-05 Avi Biopharma, Inc. Antisense antiviral compound and method for treating arenavirus infection
US7943762B2 (en) 2006-05-10 2011-05-17 Avi Biopharma, Inc. Oligonucleotide analogs having cationic intersubunit linkages
ES2373246T3 (es) 2006-08-11 2012-02-01 Prosensa Technologies B.V. Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn.
EP3034083B1 (en) 2006-09-21 2020-12-09 University of Rochester Antisense oligonucleotides for use in treating myotonic dystrophy
EP2070919B1 (en) 2006-11-24 2012-10-10 Hykes Laboratories LLC Spiroquinone compound and pharmaceutical composition
WO2009005783A1 (en) * 2007-06-28 2009-01-08 Blanchette Rockefeller Neurosciences Institute Peptides, compositions and methods for reducing beta-amyloid-mediated apoptosis
HRP20140646T1 (hr) 2007-11-15 2014-09-26 Sarepta Therapeutics, Inc. Postupak sinteze morfolino oligomera
EP2235175A4 (en) * 2007-12-20 2012-04-25 Angiochem Inc POLYPEPTIDE-NUCLEIC ACID CONJUGATES AND USES THEREOF
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
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
SI3305302T1 (sl) 2009-06-17 2018-12-31 Biogen Ma Inc. Sestave in metode za modulacijo združevanja smn2 pri subjektu

Patent Citations (4)

* 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
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2009144481A2 (en) * 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
WO2011143608A1 (en) * 2010-05-13 2011-11-17 Avi Biopharma, Inc. Antisense modulation of interleukins 17 and 23 signaling

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Lebleu et al, "Cell penetrating peptide conjugates of steric block oligonucleotides", Advanced Drug Delivery Reviews (2008), Vol 60, No. 4-5, Pages 517-529 *
Richard et al, "Cell-Penetrating Peptides - A Reevaluation of the Mechanism of Cellular Uptake", The Journal of Biological Chemistry (2003), Vol. 278, No. 1, Pages 585-590 *
Yin et al, "Effective Exon Skipping and Restoration of Dystrophin Expression by Peptide Nucleic Acid Antisense Oligonucleotides in mdx Mice", Molecular Therapy (2008), Vol. 16, No. 1, Pages 38-45 *
Zhang et al, Nuclear Medicine and Biology (2006), Vol. 33, Pages 263-269 *

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