MA42695A - Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale - Google Patents

Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale

Info

Publication number
MA42695A
MA42695A MA042695A MA42695A MA42695A MA 42695 A MA42695 A MA 42695A MA 042695 A MA042695 A MA 042695A MA 42695 A MA42695 A MA 42695A MA 42695 A MA42695 A MA 42695A
Authority
MA
Morocco
Prior art keywords
exons
inclusion
muscle atrophy
antisense oligomers
modified antisense
Prior art date
Application number
MA042695A
Other languages
English (en)
French (fr)
Inventor
Gunnar J Hanson
Marco A Passini
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 MA42695A publication Critical patent/MA42695A/fr

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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (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)
MA042695A 2015-08-28 2016-08-26 Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale MA42695A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562211678P 2015-08-28 2015-08-28
US201662379696P 2016-08-25 2016-08-25

Publications (1)

Publication Number Publication Date
MA42695A true MA42695A (fr) 2018-07-04

Family

ID=56883867

Family Applications (1)

Application Number Title Priority Date Filing Date
MA042695A MA42695A (fr) 2015-08-28 2016-08-26 Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale

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
US11555189B2 (en) 2017-10-18 2023-01-17 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
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

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
US6784291B2 (en) 2000-05-04 2004-08-31 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
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
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
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
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
KR20200133284A (ko) * 2010-05-28 2020-11-26 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
US9518259B2 (en) * 2010-06-15 2016-12-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CA3092114A1 (en) 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2012178122A2 (en) 2011-06-23 2012-12-27 Cold Spring Harbor Laboratory Phenocopy model of disease
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
WO2013086207A1 (en) 2011-12-06 2013-06-13 The Ohio State University Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
CN104411831B (zh) 2012-03-20 2020-08-11 萨勒普塔医疗公司 寡核苷酸类似物的硼酸缀合物
SG11201407483YA (en) 2012-05-16 2014-12-30 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
EP2983676B1 (en) 2013-04-12 2019-03-20 The Curators of the University of Missouri Smn2 element 1 antisense compositions and methods and uses thereof
US9988626B2 (en) * 2013-07-29 2018-06-05 Universität Zu Köln Neurocalcin delta inhibitors and therapeutic and non-therapeutic uses thereof
US20170051277A1 (en) 2013-09-13 2017-02-23 The University Of Western Australia Antisense oligomers and methods for treating smn-related pathologies
WO2015120450A1 (en) 2014-02-10 2015-08-13 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
JP2018525015A (ja) 2018-09-06
JP6987041B2 (ja) 2021-12-22
EP3341480A1 (en) 2018-07-04
AU2016317667A1 (en) 2018-03-22
WO2017040271A1 (en) 2017-03-09
US20250241940A1 (en) 2025-07-31
US10905709B2 (en) 2021-02-02
US12121532B2 (en) 2024-10-22
HK1257498A1 (zh) 2019-10-25
CA2996164A1 (en) 2017-03-09
US20210169918A1 (en) 2021-06-10
US20190015440A1 (en) 2019-01-17
JP2021048877A (ja) 2021-04-01

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