AU2016317667A1 - Modified antisense oligomers for exon inclusion in spinal muscular atrophy - Google Patents

Modified antisense oligomers for exon inclusion in spinal muscular atrophy Download PDF

Info

Publication number
AU2016317667A1
AU2016317667A1 AU2016317667A AU2016317667A AU2016317667A1 AU 2016317667 A1 AU2016317667 A1 AU 2016317667A1 AU 2016317667 A AU2016317667 A AU 2016317667A AU 2016317667 A AU2016317667 A AU 2016317667A AU 2016317667 A1 AU2016317667 A1 AU 2016317667A1
Authority
AU
Australia
Prior art keywords
pmo
alkyl
pct
apn
formula
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
Application number
AU2016317667A
Other languages
English (en)
Inventor
Gunnar J. Hanson
Marco A. Passini
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sarepta Therapeutics Inc
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
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of AU2016317667A1 publication Critical patent/AU2016317667A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
AU2016317667A 2015-08-28 2016-08-26 Modified antisense oligomers for exon inclusion in spinal muscular atrophy Abandoned AU2016317667A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562211678P 2015-08-28 2015-08-28
US62/211,678 2015-08-28
US201662379696P 2016-08-25 2016-08-25
US62/379,696 2016-08-25
PCT/US2016/048965 WO2017040271A1 (en) 2015-08-28 2016-08-26 Modified antisense oligomers for exon inclusion in spinal muscular atrophy

Publications (1)

Publication Number Publication Date
AU2016317667A1 true AU2016317667A1 (en) 2018-03-22

Family

ID=56883867

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2016317667A Abandoned AU2016317667A1 (en) 2015-08-28 2016-08-26 Modified antisense oligomers for exon inclusion in spinal muscular atrophy

Country Status (8)

Country Link
US (3) US10905709B2 (enExample)
EP (1) EP3341480A1 (enExample)
JP (2) JP6987041B2 (enExample)
AU (1) AU2016317667A1 (enExample)
CA (1) CA2996164A1 (enExample)
HK (1) HK1257498A1 (enExample)
MA (1) MA42695A (enExample)
WO (1) WO2017040271A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
EP3697910A4 (en) * 2017-10-18 2021-07-14 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMER COMPOUNDS
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
JP7446443B2 (ja) 2020-02-28 2024-03-08 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｓｍｎ２を調節するための化合物及び方法

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2802206B1 (fr) 1999-12-14 2005-04-22 Sod Conseils Rech Applic Derives de 4-aminopiperidine et leur utilisation en tant que medicament
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
EP3470072A1 (en) * 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
SI2735568T1 (en) 2006-05-10 2018-01-31 Sarepta Therapeutics, Inc. Analogues of the oligonucleotide, with cationic links between subunits
DK2049664T3 (da) 2006-08-11 2012-01-02 Prosensa Technologies Bv Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
KR20120093138A (ko) * 2009-06-17 2012-08-22 콜드스프링하버러보러토리 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
JP5585822B2 (ja) 2010-05-11 2014-09-10 東レ・ファインケミカル株式会社 光学活性ニペコチン酸誘導体の製造方法
NZ603606A (en) * 2010-05-28 2015-06-26 Sarepta Therapeutics Inc Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
EP2582397A4 (en) 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
JP6478632B2 (ja) * 2011-05-05 2019-03-06 サレプタ セラピューティクス， インコーポレイテッド ペプチドオリゴヌクレオチドコンジュゲート
AU2012272656A1 (en) 2011-06-23 2014-02-06 Cold Spring Harbor Laboratory Phenocopy model of disease
WO2013082548A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
US9944926B2 (en) * 2011-11-30 2018-04-17 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
EP2788087A4 (en) 2011-12-06 2015-08-26 Ohio State Innovation Foundation Non-ionic, low-osmolar contrast agents for the administration of antisense oligonucleotides and the treatment of diseases
WO2013142087A1 (en) 2012-03-20 2013-09-26 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
WO2013173638A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
US20160002624A1 (en) * 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
WO2014110291A1 (en) * 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
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
US9885040B2 (en) * 2013-04-12 2018-02-06 The Curators Of The University Of Missouri SMN2 element 1 antisense compositions and methods and uses thereof
EP3027753B1 (en) * 2013-07-29 2018-09-12 Universität zu Köln Neurocalcin delta inhibitors and therapeutic and non-therapeutic uses thereof
EP3044317A1 (en) * 2013-09-13 2016-07-20 The University Of Western Australia Antisense oligomers and methods for treating smn-related pathologies
US9845469B2 (en) * 2014-02-10 2017-12-19 Ohio State Innovation Foundation Antisense oligonucleotides for treatment of spinal muscular atrophy
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy

Also Published As

Publication number Publication date
HK1257498A1 (zh) 2019-10-25
MA42695A (fr) 2018-07-04
US10905709B2 (en) 2021-02-02
JP2021048877A (ja) 2021-04-01
US20190015440A1 (en) 2019-01-17
US12121532B2 (en) 2024-10-22
WO2017040271A1 (en) 2017-03-09
CA2996164A1 (en) 2017-03-09
JP6987041B2 (ja) 2021-12-22
EP3341480A1 (en) 2018-07-04
US20250241940A1 (en) 2025-07-31
JP2018525015A (ja) 2018-09-06
US20210169918A1 (en) 2021-06-10

Similar Documents

Publication Publication Date Title
AU2023203112B2 (en) Peptide oligonucleotide conjugates
JP7008056B2 (ja) 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
AU2016317667A1 (en) Modified antisense oligomers for exon inclusion in spinal muscular atrophy
JP2018530560A5 (enExample)
EP2828395B1 (en) Boronic acid conjugates of oligonucleotide analogues
TW201840339A (zh) 用於肌肉萎縮症之外顯子跳躍寡聚物結合物
JP2009515823A5 (enExample)
WO2018056871A1 (ru) Аналоги олигонуклеотидов как средства коррекции сплайсинга для лечения мышечной дистрофии дюшена
CA3122281A1 (en) Exon skipping oligomer conjugates for muscular dystrophy
JP2025038098A (ja) 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
HK40098027A (en) Exon skipping oligomers for muscular dystrophy
KR20230128689A (ko) 핵산 복합체를 포함하는 알츠하이머병의 예방 또는 치료용 조성물
HK1250922B (zh) 肽寡核苷酸缀合物
EP4219717A2 (en) Exon skipping oligomers for muscular dystrophy
DEVARAJ et al. Pd-1 Specific Aptamers
HK1206064B (en) Boronic acid conjugates of oligonucleotide analogues

Legal Events

Date Code Title Description
MK5 Application lapsed section 142(2)(e) - patent request and compl. specification not accepted