IL273838B - Peptide and oligonucleotide conjugates, preparations containing them and their uses - Google Patents

Peptide and oligonucleotide conjugates, preparations containing them and their uses

Info

Publication number
IL273838B
IL273838B IL273838A IL27383820A IL273838B IL 273838 B IL273838 B IL 273838B IL 273838 A IL273838 A IL 273838A IL 27383820 A IL27383820 A IL 27383820A IL 273838 B IL273838 B IL 273838B
Authority
IL
Israel
Prior art keywords
conjugate
alkyl
occurrence
independently
amino acid
Prior art date
Application number
IL273838A
Other languages
English (en)
Hebrew (he)
Other versions
IL273838A (en
Original Assignee
Sarepta Therapeutics 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
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of IL273838A publication Critical patent/IL273838A/en
Publication of IL273838B publication Critical patent/IL273838B/en

Links

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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (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)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (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)
IL273838A 2011-05-05 2011-11-17 Peptide and oligonucleotide conjugates, preparations containing them and their uses IL273838B (en)

Applications Claiming Priority (3)

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
US13/107,528 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

Publications (2)

Publication Number Publication Date
IL273838A IL273838A (en) 2020-05-31
IL273838B true IL273838B (en) 2022-09-01

Family

ID=45218873

Family Applications (2)

Application Number Title Priority Date Filing Date
IL273838A IL273838B (en) 2011-05-05 2011-11-17 Peptide and oligonucleotide conjugates, preparations containing them and their uses
IL229227A IL229227B (en) 2011-05-05 2013-11-04 Peptide and oligonucleotide conjugates, preparations containing them and their uses

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL229227A IL229227B (en) 2011-05-05 2013-11-04 Peptide and oligonucleotide conjugates, preparations containing them and their uses

Country Status (8)

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

Families Citing this family (92)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
SI2049664T1 (sl) 2006-08-11 2012-04-30 Prosensa Technologies Bv Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
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
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
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
HK1201514A1 (en) * 2011-11-18 2015-09-04 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
JP6496549B2 (ja) 2011-11-30 2019-04-03 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誘発されたエクソン包含
US10066228B2 (en) 2011-11-30 2018-09-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
ES2935606T3 (es) 2011-12-08 2023-03-08 Sarepta Therapeutics Inc Análogos de oligonucleótidos dirigidos a LMNA humana
NZ627896A (en) 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
LT2841578T (lt) 2012-04-23 2017-09-25 Biomarin Technologies B.V. Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui
HK1216902A1 (zh) 2012-12-20 2016-12-09 Sarepta Therapeutics, Inc. 用作治疗肌肉萎缩的改进外显子跳跃组合物
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
CN110218727A (zh) * 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
US20140315977A1 (en) 2013-03-14 2014-10-23 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
DK3015467T3 (da) 2013-05-24 2025-02-10 Ajinomoto Kk Morpholino-oligonukleotidfremstillingsfremgangsmåde
US11236338B2 (en) * 2013-09-05 2022-02-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EA030615B1 (ru) 2013-12-24 2018-08-31 Сентисс Фарма Прайвет Лимитед Офтальмологический раствор бримонидина для местного применения
DK3118311T3 (en) * 2014-03-12 2019-03-11 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
CA2948373A1 (en) 2014-05-16 2015-11-19 Oregon State University Antisense antibacterial compounds and methods
CA2948568A1 (en) 2014-05-19 2015-11-26 David Greenberg Antisense antibacterial compounds and methods
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
EP3240794A4 (en) 2014-12-31 2018-10-31 Oregon State University Antisense antibacterial compounds and methods
EP3262056A4 (en) * 2015-02-27 2018-09-19 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
MA41795A (fr) * 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
AU2016264471B2 (en) * 2015-05-19 2022-01-27 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
AU2016302009B2 (en) * 2015-08-05 2021-09-30 Eisai R&D Management Co., Ltd. Chiral reagents for preparation of homogeneous oligomers
US10905709B2 (en) * 2015-08-28 2021-02-02 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
EP3858993A1 (en) 2015-10-09 2021-08-04 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
AU2016379399B2 (en) 2015-12-23 2022-12-08 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
US11142764B2 (en) 2015-12-23 2021-10-12 Board Of Regents, The University Of Texas System 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
MX2018013157A (es) 2016-04-29 2019-05-23 Sarepta Therapeutics Inc Analogos de oligonucleotidos dirigidos a lmna humana.
KR102523522B1 (ko) * 2016-06-30 2023-04-20 사렙타 쎄러퓨틱스 인코퍼레이티드 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법
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
KR102810425B1 (ko) * 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
PT3554552T (pt) * 2016-12-19 2022-10-03 Sarepta Therapeutics Inc Conjugados de oligómero de skipping de exão para distrofia muscular
KR20240006023A (ko) * 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
US20200131231A1 (en) 2017-02-17 2020-04-30 Oxford University Innovation Limited Cell penetrating peptides
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
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
EA201991450A1 (ru) * 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
WO2019067979A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
EP3687547A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
WO2019067981A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
WO2019079386A1 (en) 2017-10-17 2019-04-25 Sarepta Therapeutics, Inc. CELL PENETRATION PEPTIDES FOR ANTISENSE ADMINISTRATION
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
EP3765030A4 (en) 2018-03-16 2022-01-26 Sarepta Therapeutics, Inc. CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION
JP2021521794A (ja) * 2018-04-26 2021-08-30 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およびオリゴマーコンジュゲート
EP3806868A4 (en) 2018-06-13 2022-06-22 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
JP7564087B2 (ja) * 2018-07-30 2024-10-08 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達のための三量体ペプチド
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812980D0 (en) * 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
US12465646B2 (en) * 2018-12-07 2025-11-11 Oxford University Innovation Limited Linkers
IL318474A (en) 2018-12-13 2025-03-01 Sarepta Therapeutics Inc Axon-skipping oligomer complexes for muscular dystrophy
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
WO2020214763A1 (en) 2019-04-18 2020-10-22 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
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
WO2021167107A1 (ja) 2020-02-22 2021-08-26 Ｊｃｒファーマ株式会社 ヒトトランスフェリンレセプター結合ペプチド
IL295967A (en) 2020-02-28 2022-10-01 Nippon Shinyaku Co Ltd Antisense nucleic acids for splicing on exon 51
CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
WO2022061216A1 (en) * 2020-09-21 2022-03-24 Icahn School Of Medicine At Mount Sinai Archaea l30 proteins as universal influenza virus therapeutics
EP4253540A4 (en) * 2020-11-30 2025-09-10 Univ Niigata ANTISENSE NUCLEIC ACID AND ITS USE
EP4267191A1 (en) 2020-12-23 2023-11-01 Sarepta Therapeutics, Inc. Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy
WO2022171972A1 (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
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
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
CA3225573A1 (en) 2021-07-08 2023-01-12 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
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
WO2023027125A1 (ja) 2021-08-24 2023-03-02 ペプチドリーム株式会社 ヒトトランスフェリンレセプター結合抗体－ペプチドコンジュゲート
WO2023055774A1 (en) 2021-09-30 2023-04-06 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
CN119013401A (zh) 2022-03-17 2024-11-22 萨勒普塔医疗公司 二氨基磷酸酯吗啉代寡聚物缀合物
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
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

Family Cites Families (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
WO1986005519A1 (en) 1985-03-15 1986-09-25 James Summerton Polynucleotide assay reagent 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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
AU659482B2 (en) 1991-06-28 1995-05-18 Massachusetts Institute Of Technology Localized oligonucleotide therapy
ES2123062T3 (es) 1992-08-21 1999-01-01 Biogen Inc Polipeptidos de transporte derivados de la proteina tat.
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
CN1263473A (zh) 1997-05-21 2000-08-16 利兰·斯坦福青年大学托管委员会 增加跨生物膜转运的组合物和方法
AU741546B2 (en) * 1997-07-24 2001-12-06 Perseptive Biosystems, Inc. Conjugates of transporter peptides and nucleic acid analogs, and their use
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
DE60233137D1 (de) 2001-02-16 2009-09-10 Univ R Transporter mit beabstandeten arginin-teilchen
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
US7468418B2 (en) 2003-04-29 2008-12-23 Avi Biopharma., Inc. Compositions for enhancing transport of molecules into cells
CA2533218C (en) 2003-08-05 2014-11-18 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
JP2007536253A (ja) * 2004-05-04 2007-12-13 ナステック・ファーマシューティカル・カンパニー・インコーポレーテッド 細胞内への核酸の送達を増強し、細胞中の標的遺伝子の発現を修飾するための組成物及び方法
US20050288246A1 (en) * 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
EP2933332A1 (en) 2004-06-28 2015-10-21 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
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
WO2007030576A2 (en) 2005-09-08 2007-03-15 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
US7582615B2 (en) 2006-03-07 2009-09-01 Avi Biopharma, Inc. Antisense antiviral compound and method for treating arenavirus infection
PL2735568T3 (pl) 2006-05-10 2018-02-28 Sarepta Therapeutics, Inc. Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami
SI2049664T1 (sl) 2006-08-11 2012-04-30 Prosensa Technologies Bv Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev
EP2560001B1 (en) 2006-09-21 2016-04-13 University of Rochester Compositions and methods related to protein displacement therapy for myotonic distrophy
US8119686B2 (en) 2006-11-24 2012-02-21 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
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2009005793A2 (en) * 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
RU2606627C2 (ru) 2007-11-15 2017-01-10 Серепта Терапьютикс,Инк. Способ синтеза морфолиновых олигомеров
BRPI0821310A2 (pt) * 2007-12-20 2015-06-16 Angiochem Inc Conjugados polipeptídeos-ácido nucleico e usos dos mesmos
WO2009144481A2 (en) * 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
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
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
CA3090304A1 (en) * 2010-05-13 2011-11-17 Sarepta Therapeutics, Inc. Antisense modulation of interleukins 17 and 23 signaling

Also Published As

Publication number Publication date
CA2834128A1 (en) 2012-11-08
JP6478632B2 (ja) 2019-03-06
JP2018135396A (ja) 2018-08-30
AU2023203112B2 (en) 2025-06-05
AU2017206179A1 (en) 2017-08-03
AU2023203112A1 (en) 2023-06-08
CN103619356B (zh) 2017-09-12
KR102229650B1 (ko) 2021-03-19
KR20140028058A (ko) 2014-03-07
JP2014515762A (ja) 2014-07-03
KR102339196B1 (ko) 2021-12-15
AU2025226744A1 (en) 2025-09-25
AU2011367230B2 (en) 2017-08-10
EP2704749A1 (en) 2014-03-12
AU2019204913A1 (en) 2019-07-25
CA3092114A1 (en) 2012-11-08
AU2011367230A1 (en) 2013-12-05
JP2024032974A (ja) 2024-03-12
AU2021202224A1 (en) 2021-05-06
JP2016185991A (ja) 2016-10-27
JP6884250B2 (ja) 2021-06-09
CN107693797B (zh) 2021-05-11
JP2021113232A (ja) 2021-08-05
IL229227A0 (en) 2014-01-30
WO2012150960A1 (en) 2012-11-08
JP2020111607A (ja) 2020-07-27
CN107693797A (zh) 2018-02-16
IL273838A (en) 2020-05-31
KR102183273B1 (ko) 2020-11-27
KR20210032545A (ko) 2021-03-24
KR20190084351A (ko) 2019-07-16
CN103619356A (zh) 2014-03-05
IL229227B (en) 2020-07-30

Similar Documents

Publication Publication Date Title
AU2023203112B2 (en) Peptide oligonucleotide conjugates
US11732259B2 (en) Peptide oligonucleotide conjugates
US10760078B2 (en) Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
AU2013202731B2 (en) Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
NZ761522B2 (en) Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups