HK1206064A1 - 寡核苷酸類似物的硼酸綴合物 - Google Patents

寡核苷酸類似物的硼酸綴合物

Info

Publication number
HK1206064A1
HK1206064A1 HK15106455.8A HK15106455A HK1206064A1 HK 1206064 A1 HK1206064 A1 HK 1206064A1 HK 15106455 A HK15106455 A HK 15106455A HK 1206064 A1 HK1206064 A1 HK 1206064A1
Authority
HK
Hong Kong
Prior art keywords
boronic acid
acid conjugates
oligonucleotide analogues
analogues
oligonucleotide
Prior art date
Application number
HK15106455.8A
Other languages
English (en)
Inventor
Gunnar J Hanson
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 HK1206064A1 publication Critical patent/HK1206064A1/zh

Links

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6596Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having atoms other than oxygen, sulfur, selenium, tellurium, nitrogen or phosphorus as ring hetero atoms
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558Heterocyclic 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
    • C07F9/65583Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/331Universal or degenerate base
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
HK15106455.8A 2012-03-20 2015-07-07 寡核苷酸類似物的硼酸綴合物 HK1206064A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261613385P 2012-03-20 2012-03-20
PCT/US2013/029684 WO2013142087A1 (en) 2012-03-20 2013-03-07 Boronic acid conjugates of oligonucleotide analogues

Publications (1)

Publication Number Publication Date
HK1206064A1 true HK1206064A1 (zh) 2015-12-31

Family

ID=49223176

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15106455.8A HK1206064A1 (zh) 2012-03-20 2015-07-07 寡核苷酸類似物的硼酸綴合物

Country Status (15)

Country Link
US (1) US9920085B2 (zh)
EP (1) EP2828395B1 (zh)
JP (1) JP6542662B2 (zh)
KR (1) KR102079284B1 (zh)
CN (1) CN104411831B (zh)
AU (3) AU2013235691B2 (zh)
BR (1) BR112014023347A2 (zh)
CA (1) CA2868174A1 (zh)
EA (1) EA031110B1 (zh)
ES (1) ES2706198T3 (zh)
HK (1) HK1206064A1 (zh)
IN (1) IN2014DN08451A (zh)
MX (1) MX358497B (zh)
NZ (1) NZ700399A (zh)
WO (1) WO2013142087A1 (zh)

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KR101958491B1 (ko) 2009-11-12 2019-03-15 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
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
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
US9278987B2 (en) 2011-11-18 2016-03-08 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
TR201903009T4 (tr) * 2013-03-14 2019-03-21 Sarepta Therapeutics Inc Musküler distrofi tedavisine yönelik ekson atlama bileşimleri.
BR112015023001B8 (pt) * 2013-03-14 2022-08-09 Sarepta Therapeutics Inc Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
WO2014144978A2 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
US10675356B2 (en) * 2015-05-19 2020-06-09 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP3302489A4 (en) * 2015-06-04 2019-02-06 Sarepta Therapeutics, Inc. METHOD AND COMPOUNDS FOR THE TREATMENT OF DISEASES AND SUFFERING IN CONNECTION WITH LYMPHOCYTES
EP3341480A1 (en) 2015-08-28 2018-07-04 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
AU2016335032B2 (en) * 2015-10-05 2022-04-14 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion

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Also Published As

Publication number Publication date
AU2017202883A1 (en) 2017-05-18
ES2706198T3 (es) 2019-03-27
JP2015512254A (ja) 2015-04-27
EP2828395B1 (en) 2018-10-24
NZ700399A (en) 2016-07-29
KR102079284B1 (ko) 2020-02-19
EA201491726A1 (ru) 2015-03-31
JP6542662B2 (ja) 2019-07-10
EP2828395A4 (en) 2015-11-18
EP2828395A1 (en) 2015-01-28
EA031110B1 (ru) 2018-11-30
US9920085B2 (en) 2018-03-20
CN104411831B (zh) 2020-08-11
AU2013235691B2 (en) 2017-02-02
CA2868174A1 (en) 2013-09-26
IN2014DN08451A (zh) 2015-05-08
MX2014011276A (es) 2014-10-06
US20150080340A1 (en) 2015-03-19
AU2013235691A1 (en) 2014-10-16
AU2019201989A1 (en) 2019-04-11
WO2013142087A1 (en) 2013-09-26
BR112014023347A2 (pt) 2017-07-18
CN104411831A (zh) 2015-03-11
KR20140138995A (ko) 2014-12-04
MX358497B (es) 2018-08-23

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PC Patent ceased (i.e. patent has lapsed due to the failure to pay the renewal fee)

Effective date: 20230305