HK1257498A1 - 在脊髓性肌肉萎缩症中用於内显子纳入的改良的反义寡聚体 - Google Patents

在脊髓性肌肉萎缩症中用於内显子纳入的改良的反义寡聚体 Download PDF

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Publication number
HK1257498A1
HK1257498A1 HK18116288.7A HK18116288A HK1257498A1 HK 1257498 A1 HK1257498 A1 HK 1257498A1 HK 18116288 A HK18116288 A HK 18116288A HK 1257498 A1 HK1257498 A1 HK 1257498A1
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HK
Hong Kong
Prior art keywords
muscular atrophy
spinal muscular
antisense oligomers
modified antisense
exon inclusion
Prior art date
Application number
HK18116288.7A
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English (en)
Chinese (zh)
Inventor
Marco A. Passini
Gunnar J. Hanson
Original Assignee
Sarepta Therapeutics, Inc.
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Publication date
Application filed by Sarepta Therapeutics, Inc. filed Critical Sarepta Therapeutics, Inc.
Publication of HK1257498A1 publication Critical patent/HK1257498A1/zh

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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Psychology (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)
HK18116288.7A 2015-08-28 2016-08-26 在脊髓性肌肉萎缩症中用於内显子纳入的改良的反义寡聚体 HK1257498A1 (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562211678P 2015-08-28 2015-08-28
US201562211678P 2015-08-28
US201662379696P 2016-08-25 2016-08-25
US201662379696P 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
HK1257498A1 true HK1257498A1 (zh) 2019-10-25

Family

ID=56883867

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18116288.7A HK1257498A1 (zh) 2015-08-28 2016-08-26 在脊髓性肌肉萎缩症中用於内显子纳入的改良的反义寡聚体

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
MA42695A (fr) 2015-08-28 2018-07-04 Sarepta Therapeutics Inc Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale
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
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

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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
US8361977B2 (en) * 2005-06-23 2013-01-29 Isis Pharmaceuticals, 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
ES2657400T3 (es) 2006-05-10 2018-03-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
JP6047270B2 (ja) 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー．フェー． Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
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
PT3449926T (pt) * 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
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
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 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
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
WO2012150960A1 (en) 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
AU2012272656A1 (en) * 2011-06-23 2014-02-06 Cold Spring Harbor Laboratory Phenocopy model of disease
EP2785729B1 (en) 2011-11-30 2020-11-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
AU2012345638C1 (en) * 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
US9725716B2 (en) * 2011-12-06 2017-08-08 Ohio State Innovation Foundation and Research Institute at Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
CA2868174A1 (en) 2012-03-20 2013-09-26 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
SG11201407483YA (en) * 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
WO2013173789A2 (en) * 2012-05-17 2013-11-21 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
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
EP3560502B1 (en) * 2013-04-12 2025-09-24 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
EP3044317A1 (en) * 2013-09-13 2016-07-20 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
MA42695A (fr) 2015-08-28 2018-07-04 Sarepta Therapeutics Inc Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale

Also Published As

Publication number Publication date
US20210169918A1 (en) 2021-06-10
US12121532B2 (en) 2024-10-22
US20250241940A1 (en) 2025-07-31
MA42695A (fr) 2018-07-04
JP6987041B2 (ja) 2021-12-22
AU2016317667A1 (en) 2018-03-22
US10905709B2 (en) 2021-02-02
CA2996164A1 (en) 2017-03-09
US20190015440A1 (en) 2019-01-17
JP2021048877A (ja) 2021-04-01
EP3341480A1 (en) 2018-07-04
JP2018525015A (ja) 2018-09-06
WO2017040271A1 (en) 2017-03-09

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