EP3463389A4 - Processes for preparing phosphorodiamidate morpholino oligomers - Google Patents

Processes for preparing phosphorodiamidate morpholino oligomers Download PDF

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Publication number
EP3463389A4
EP3463389A4 EP17803760.2A EP17803760A EP3463389A4 EP 3463389 A4 EP3463389 A4 EP 3463389A4 EP 17803760 A EP17803760 A EP 17803760A EP 3463389 A4 EP3463389 A4 EP 3463389A4
Authority
EP
European Patent Office
Prior art keywords
processes
morpholino oligomers
phosphorodiamidate morpholino
preparing
preparing phosphorodiamidate
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.)
Pending
Application number
EP17803760.2A
Other languages
German (de)
French (fr)
Other versions
EP3463389A2 (en
Inventor
Bao Cai
Mitchell MARTINI
Katie THOMAS
Ross SHIMABUKU
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 EP3463389A2 publication Critical patent/EP3463389A2/en
Publication of EP3463389A4 publication Critical patent/EP3463389A4/en
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic System
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65583—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system each of the hetero rings containing nitrogen as ring hetero atom
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic System
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • C07F9/65616—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Polyethers (AREA)
  • Macromolecular Compounds Obtained By Forming Nitrogen-Containing Linkages In General (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP17803760.2A 2016-06-30 2017-06-30 Processes for preparing phosphorodiamidate morpholino oligomers Pending EP3463389A4 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662357153P 2016-06-30 2016-06-30
US201662357134P 2016-06-30 2016-06-30
US201762508256P 2017-05-18 2017-05-18
PCT/US2017/040311 WO2017205879A2 (en) 2016-05-24 2017-06-30 Processes for preparing phosphorodiamidate morpholino oligomers

Publications (2)

Publication Number Publication Date
EP3463389A2 EP3463389A2 (en) 2019-04-10
EP3463389A4 true EP3463389A4 (en) 2020-02-19

Family

ID=64425889

Family Applications (1)

Application Number Title Priority Date Filing Date
EP17803760.2A Pending EP3463389A4 (en) 2016-06-30 2017-06-30 Processes for preparing phosphorodiamidate morpholino oligomers

Country Status (10)

Country Link
EP (1) EP3463389A4 (en)
JP (1) JP7022079B2 (en)
KR (1) KR102523522B1 (en)
CN (1) CN109152792B (en)
AU (1) AU2017270598B2 (en)
CA (1) CA3024153A1 (en)
CO (1) CO2018013831A2 (en)
MX (1) MX2018014160A (en)
SG (1) SG11201809502YA (en)
WO (1) WO2017205879A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI541024B (en) 2010-09-01 2016-07-11 日本新藥股份有限公司 Antisense nucleic acid
JP7022078B2 (en) * 2016-06-30 2022-02-17 サレプタ セラピューティクス， インコーポレイテッド Process for preparing phosphorodiamidart morpholino oligomers
EP3657604A1 (en) 2018-11-26 2020-05-27 TE Connectivity Germany GmbH Cable terminating assembly with electrically insulating cutting blades
EP4230196A1 (en) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Compounds for use in the treatment of dystrophinopathies

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011150408A2 (en) * 2010-05-28 2011-12-01 Avi Biopharma, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
WO2013082551A1 (en) * 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
WO2014153240A2 (en) * 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy

Family Cites Families (23)

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Publication number Priority date Publication date Assignee Title
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
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. Polynucleotide assay reagent and method
CA1339987C (en) 1988-09-01 1998-08-04 Robert Bruce Merrifield Peptide synthesis method and solid support for use in the method
JP3572617B2 (en) * 1993-12-29 2004-10-06 藤沢薬品工業株式会社 Pyrazolopyridine adenosine antagonist
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
CA2704261C (en) 2007-11-15 2015-05-26 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
US9050373B2 (en) * 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
TWI541024B (en) * 2010-09-01 2016-07-11 日本新藥股份有限公司 Antisense nucleic acid
ES2607603T3 (en) 2010-09-30 2017-04-03 Nippon Shinyaku Co., Ltd. Morpholino nucleic acid derivative
WO2012150960A1 (en) * 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
CN105378081B (en) 2013-03-14 2019-06-14 萨勒普塔医疗公司 For treating the exon skipping composition of muscular dystrophy
EP3015467A4 (en) * 2013-05-24 2016-11-02 Ajinomoto Kk Morpholino oligonucleotide manufacturing method
AU2017269355B2 (en) * 2016-05-24 2021-04-15 Sarepta Therapeutics, Inc. Processes for preparing oligomers
WO2017205513A1 (en) * 2016-05-24 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
SG11201810143PA (en) 2016-06-30 2019-01-30 Sarepta Therapeutics Inc Exon skipping oligomers for muscular dystrophy
JP7022078B2 (en) 2016-06-30 2022-02-17 サレプタ セラピューティクス， インコーポレイテッド Process for preparing phosphorodiamidart morpholino oligomers

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011150408A2 (en) * 2010-05-28 2011-12-01 Avi Biopharma, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
WO2013082551A1 (en) * 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
WO2014153240A2 (en) * 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
See also references of WO2017205879A2 *
VIRTA P ET AL: "Solid-supported synthesis of oligomeric bioconjugates", TETRAHE, ELSEVIER SCIENCE PUBLISHERS, AMSTERDAM, NL, vol. 59, no. 28, 7 July 2003 (2003-07-07), pages 5137 - 5174, XP004434882, ISSN: 0040-4020, DOI: 10.1016/S0040-4020(03)00704-X *

Also Published As

Publication number Publication date
KR20190020674A (en) 2019-03-04
CN109152792B (en) 2021-09-14
CO2018013831A2 (en) 2018-12-28
KR102523522B1 (en) 2023-04-20
JP7022079B2 (en) 2022-02-17
CN109152792A (en) 2019-01-04
WO2017205879A2 (en) 2017-11-30
SG11201809502YA (en) 2018-12-28
AU2017270598B2 (en) 2022-12-01
CA3024153A1 (en) 2017-11-30
EP3463389A2 (en) 2019-04-10
JP2019518748A (en) 2019-07-04
AU2017270598A1 (en) 2019-01-17
MX2018014160A (en) 2019-04-01
WO2017205879A3 (en) 2018-01-18

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