EP3463389A4 - Procédés de préparation d'oligomères morpholino de phosphorodiamidate - Google Patents

Procédés de préparation d'oligomères morpholino de phosphorodiamidate 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
producing
morpholino oligomers
phosphorodiamidate morpholino
phosphorodiamidate
oligomers
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)
English (en)
Other versions
EP3463389A2 (fr
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/fr
Publication of EP3463389A4 publication Critical patent/EP3463389A4/fr
Pending legal-status Critical Current

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    • 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 Table
    • 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 Table
    • 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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Polyethers (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Macromolecular Compounds Obtained By Forming Nitrogen-Containing Linkages In General (AREA)
EP17803760.2A 2016-06-30 2017-06-30 Procédés de préparation d'oligomères morpholino de phosphorodiamidate Pending EP3463389A4 (fr)

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 (fr) 2016-05-24 2017-06-30 Procédés de préparation d'oligomères morpholino de phosphorodiamidate

Publications (2)

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

Family

ID=64425889

Family Applications (1)

Application Number Title Priority Date Filing Date
EP17803760.2A Pending EP3463389A4 (fr) 2016-06-30 2017-06-30 Procédés de préparation d'oligomères morpholino de phosphorodiamidate

Country Status (10)

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
IL263044B2 (en) 2016-05-24 2024-06-01 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
CN109152775B (zh) * 2016-06-30 2022-04-26 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
EP4230196A1 (fr) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Composés à utiliser dans le traitement des dystrophinopathies
KR20240105985A (ko) 2022-12-29 2024-07-08 최해용 스마트폰 용 프로젝터

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011150408A2 (fr) * 2010-05-28 2011-12-01 Avi Biopharma, Inc. Analogues oligonucléotidiques ayant des liaisons modifiées entre sous-unités et/ou des groupes terminaux modifiés
WO2013082551A1 (fr) * 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Inclusion d'exon induite pour l'amyotrophie spinale
WO2014153240A2 (fr) * 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Compositions de sauts d'exon pour le traitement de la dystrophie musculaire

Family Cites Families (23)

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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
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
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
WO1986005519A1 (fr) 1985-03-15 1986-09-25 James Summerton Reactif et procede d'analyse de polynucleotides
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
DE68914843T2 (de) 1988-09-01 1994-11-10 Risoe Forskningscenter Peptid-synthese und fester träger zur verwendung dabei.
CA2180253A1 (fr) * 1993-12-29 1995-07-06 Atsushi Akahane Compose pyrazolopyridine antagoniste de l'adenosine
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
DK2207779T3 (da) 2007-11-15 2014-07-14 Sarepta Therapeutics Inc Fremgangsmåde til syntese af morpholinooligomerer
US8299206B2 (en) 2007-11-15 2012-10-30 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 (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
DK2623507T3 (da) 2010-09-30 2017-01-02 Nippon Shinyaku Co Ltd Morpholinonukleinsyrederivat
IL273838B (en) * 2011-05-05 2022-09-01 Sarepta Therapeutics Inc Peptide and oligonucleotide conjugates, preparations containing them and their uses
EA202090946A3 (ru) 2013-03-14 2021-07-30 Сарепта Терапьютикс, Инк. Композиции, обеспечивающие пропускание экзонов, для лечения мышечной дистрофии
EP3015467B1 (fr) * 2013-05-24 2024-12-04 Ajinomoto Co., Inc. Procédé de fabrication de morpholino-oligonucléotide
SG10202101830WA (en) 2016-05-24 2021-04-29 Sarepta Therapeutics Inc Processes for preparing oligomers
MX383656B (es) * 2016-05-24 2025-03-14 Sarepta Therapeutics Inc Procesos para preparar oligómeros de morfolino fosforodiamidato.
CN109152775B (zh) * 2016-06-30 2022-04-26 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
AU2017290231A1 (en) 2016-06-30 2019-02-07 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011150408A2 (fr) * 2010-05-28 2011-12-01 Avi Biopharma, Inc. Analogues oligonucléotidiques ayant des liaisons modifiées entre sous-unités et/ou des groupes terminaux modifiés
WO2013082551A1 (fr) * 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Inclusion d'exon induite pour l'amyotrophie spinale
WO2014153240A2 (fr) * 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Compositions de sauts d'exon pour le traitement de la dystrophie musculaire

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

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