CA2996164A1 - 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
CA2996164A1
CA2996164A1 CA2996164A CA2996164A CA2996164A1 CA 2996164 A1 CA2996164 A1 CA 2996164A1 CA 2996164 A CA2996164 A CA 2996164A CA 2996164 A CA2996164 A CA 2996164A CA 2996164 A1 CA2996164 A1 CA 2996164A1
Authority
CA
Canada
Prior art keywords
pmo
alkyl
formula
seq
moiety
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
CA2996164A
Other languages
English (en)
French (fr)
Inventor
Marco A. Passini
Gunnar J. Hanson
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 CA2996164A1 publication Critical patent/CA2996164A1/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)
  • 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)
CA2996164A 2015-08-28 2016-08-26 Modified antisense oligomers for exon inclusion in spinal muscular atrophy Abandoned CA2996164A1 (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
CA2996164A1 true CA2996164A1 (en) 2017-03-09

Family

ID=56883867

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2996164A Abandoned CA2996164A1 (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 (OSRAM)
EP (1) EP3341480A1 (OSRAM)
JP (2) JP6987041B2 (OSRAM)
AU (1) AU2016317667A1 (OSRAM)
CA (1) CA2996164A1 (OSRAM)
HK (1) HK1257498A1 (OSRAM)
MA (1) MA42695A (OSRAM)
WO (1) WO2017040271A1 (OSRAM)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2017040271A1 (en) 2015-08-28 2017-03-09 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
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
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
BR112022016238A2 (pt) 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular smn2

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
KR20020097241A (ko) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 스플라이스-영역 안티센스 조성물 및 방법
DK1910395T3 (da) * 2005-06-23 2013-02-04 Isis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
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.
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
SI3305302T1 (sl) * 2009-06-17 2018-12-31 Biogen Ma Inc. Sestave in metode za modulacijo združevanja smn2 pri subjektu
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
CA2799501C (en) 2010-05-28 2022-02-15 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
KR102229650B1 (ko) * 2011-05-05 2021-03-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
WO2012178122A2 (en) 2011-06-23 2012-12-27 Cold Spring Harbor Laboratory Phenocopy model of disease
ES2727481T3 (es) * 2011-11-30 2019-10-16 Sarepta Therapeutics Inc Inclusión inducida de exón en atrofia muscular espinal
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
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
EP2828395B1 (en) * 2012-03-20 2018-10-24 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
DK2850186T3 (en) * 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
WO2013173789A2 (en) 2012-05-17 2013-11-21 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
EP2943225A4 (en) * 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
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
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
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
US9845469B2 (en) * 2014-02-10 2017-12-19 Ohio State Innovation Foundation Antisense oligonucleotides for treatment of spinal muscular atrophy
WO2017040271A1 (en) 2015-08-28 2017-03-09 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy

Also Published As

Publication number Publication date
AU2016317667A1 (en) 2018-03-22
US12121532B2 (en) 2024-10-22
EP3341480A1 (en) 2018-07-04
JP6987041B2 (ja) 2021-12-22
MA42695A (fr) 2018-07-04
HK1257498A1 (zh) 2019-10-25
JP2021048877A (ja) 2021-04-01
WO2017040271A1 (en) 2017-03-09
US20190015440A1 (en) 2019-01-17
US10905709B2 (en) 2021-02-02
JP2018525015A (ja) 2018-09-06
US20250241940A1 (en) 2025-07-31
US20210169918A1 (en) 2021-06-10

Similar Documents

Publication Publication Date Title
JP6671449B2 (ja) 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含
JP7342169B2 (ja) 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法
US20250241940A1 (en) Modified antisense oligomers for exon inclusion in spinal muscular atrophy
US20220090084A1 (en) Antisense-induced exon exclusion in myostatin
BR112017018383B1 (pt) Compostos anti-sentido que induzem a inclusão de éxon2, composições farmacêuticas que compreendem ditos compostos e usos dos mesmos para tratar doença de armazenamento de glicogênio tipo ii
JP2023053352A (ja) アンチセンスオリゴマー化合物
US20240415857A1 (en) Antisense-induced exon exclusion in type vii collagen
EA042313B1 (ru) Антисмысловые олигомеры и способы их применения для лечения заболеваний, связанных с геном кислой альфа-глюкозидазы
BR112016005062B1 (pt) Compostos de oligômero antissenso, composição farmacêutica compreendendo os ditos compostos e usos dos mesmos para tratar uma doença de armazenamento de glicogênio tipo ii

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20210824

EEER Examination request

Effective date: 20210824

EEER Examination request

Effective date: 20210824

EEER Examination request

Effective date: 20210824

FZDE Discontinued

Effective date: 20240130