CN109562123A - 磷酸二酰胺吗啉代寡聚物药物组合物 - Google Patents

磷酸二酰胺吗啉代寡聚物药物组合物 Download PDF

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Publication number
CN109562123A
CN109562123A CN201780030581.5A CN201780030581A CN109562123A CN 109562123 A CN109562123 A CN 109562123A CN 201780030581 A CN201780030581 A CN 201780030581A CN 109562123 A CN109562123 A CN 109562123A
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CN
China
Prior art keywords
pharmaceutical composition
eteplirsen
sodium chloride
total volume
another embodiment
Prior art date
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Pending
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CN201780030581.5A
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English (en)
Chinese (zh)
Inventor
T.霍尔特
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SA Leputa Medical Co
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SA Leputa Medical Co
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Publication of CN109562123A publication Critical patent/CN109562123A/zh
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02Inorganic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Inorganic Chemistry (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN201780030581.5A 2016-05-24 2017-05-24 磷酸二酰胺吗啉代寡聚物药物组合物 Pending CN109562123A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662340947P 2016-05-24 2016-05-24
US62/340947 2016-05-24
US201662429160P 2016-12-02 2016-12-02
US62/429160 2016-12-02
PCT/US2017/034265 WO2017213854A1 (en) 2016-05-24 2017-05-24 Pharmaceutical composition comprising eteplirsen

Publications (1)

Publication Number Publication Date
CN109562123A true CN109562123A (zh) 2019-04-02

Family

ID=59093599

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780030581.5A Pending CN109562123A (zh) 2016-05-24 2017-05-24 磷酸二酰胺吗啉代寡聚物药物组合物

Country Status (15)

Country Link
US (1) US20190275072A1 (https=)
EP (1) EP3463390A1 (https=)
JP (1) JP2019516730A (https=)
KR (1) KR20190009343A (https=)
CN (1) CN109562123A (https=)
AU (1) AU2017278699A1 (https=)
BR (1) BR112018074299A2 (https=)
CA (1) CA3024178A1 (https=)
CO (1) CO2018013828A2 (https=)
IL (1) IL263040A (https=)
MA (1) MA45158A (https=)
MX (1) MX2018014129A (https=)
SG (1) SG11201809494VA (https=)
TW (1) TW201805002A (https=)
WO (1) WO2017213854A1 (https=)

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
EP3806868A4 (en) * 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
WO2019241385A2 (en) * 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy
JP7579147B2 (ja) * 2018-06-14 2024-11-07 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート
EP4671372A3 (en) 2024-01-29 2026-03-11 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103933549A (zh) * 2013-01-17 2014-07-23 刘海俊 一种新的血管抑素滴眼剂及其制备方法
CN105307723A (zh) * 2013-03-15 2016-02-03 萨勒普塔医疗公司 改进的用于治疗肌营养不良的组合物

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US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library 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
JP3022967B2 (ja) 1985-03-15 2000-03-21 アンチバイラルズ インコーポレイテッド 立体規則性ポリヌクレオチド結合ポリマー
EP0433345B1 (en) 1988-09-01 1994-04-20 Forskningscenter Riso Peptide synthesis method and solid support for use in the method
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
MX2010004955A (es) 2007-11-15 2010-06-30 Avi Biopharma Inc Metodo de sintesis de oligomeros de morfolina.
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
RS55610B1 (sr) 2010-09-30 2017-06-30 Nippon Shinyaku Co Ltd Derivat morfolino nukleinske kiseline
ES2727481T3 (es) 2011-11-30 2019-10-16 Sarepta Therapeutics Inc Inclusión inducida de exón en atrofia muscular espinal

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103933549A (zh) * 2013-01-17 2014-07-23 刘海俊 一种新的血管抑素滴眼剂及其制备方法
CN105307723A (zh) * 2013-03-15 2016-02-03 萨勒普塔医疗公司 改进的用于治疗肌营养不良的组合物

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
李凌等: "《生物化学与分子生物学实验指导》", 31 August 2015, 人民军医出版社 *

Also Published As

Publication number Publication date
CA3024178A1 (en) 2017-12-14
IL263040A (en) 2018-12-31
AU2017278699A1 (en) 2018-11-15
US20190275072A1 (en) 2019-09-12
BR112018074299A2 (pt) 2019-03-12
WO2017213854A1 (en) 2017-12-14
MA45158A (fr) 2019-04-10
SG11201809494VA (en) 2018-12-28
CO2018013828A2 (es) 2018-12-28
KR20190009343A (ko) 2019-01-28
EP3463390A1 (en) 2019-04-10
MX2018014129A (es) 2019-04-29
TW201805002A (zh) 2018-02-16
JP2019516730A (ja) 2019-06-20

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Application publication date: 20190402