TW201805002A - 磷醯二胺嗎啉代寡聚物醫藥組合物 - Google Patents
磷醯二胺嗎啉代寡聚物醫藥組合物 Download PDFInfo
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- TW201805002A TW201805002A TW106117190A TW106117190A TW201805002A TW 201805002 A TW201805002 A TW 201805002A TW 106117190 A TW106117190 A TW 106117190A TW 106117190 A TW106117190 A TW 106117190A TW 201805002 A TW201805002 A TW 201805002A
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- Prior art keywords
- pharmaceutical composition
- resin
- disodium hydrogen
- another embodiment
- sodium chloride
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- 229950005470 eteplirsen Drugs 0.000 claims abstract description 5
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- 239000011159 matrix material Substances 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 229910052751 metal Inorganic materials 0.000 description 1
- 239000002184 metal Substances 0.000 description 1
- 201000006938 muscular dystrophy Diseases 0.000 description 1
- 230000003472 neutralizing effect Effects 0.000 description 1
- 239000002547 new drug Substances 0.000 description 1
- 229920001220 nitrocellulos Polymers 0.000 description 1
- 239000002777 nucleoside Substances 0.000 description 1
- 150000003833 nucleoside derivatives Chemical class 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 229920001778 nylon Polymers 0.000 description 1
- 230000003287 optical effect Effects 0.000 description 1
- 238000004806 packaging method and process Methods 0.000 description 1
- 208000035139 partial with pericentral spikes epilepsy Diseases 0.000 description 1
- 230000002688 persistence Effects 0.000 description 1
- 239000011129 pharmaceutical packaging material Substances 0.000 description 1
- 229940127557 pharmaceutical product Drugs 0.000 description 1
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 description 1
- 229920001083 polybutene Polymers 0.000 description 1
- 239000005017 polysaccharide Substances 0.000 description 1
- 229920001282 polysaccharide Polymers 0.000 description 1
- 229920002635 polyurethane Polymers 0.000 description 1
- 239000004814 polyurethane Substances 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- UIDUKLCLJMXFEO-UHFFFAOYSA-N propylsilane Chemical compound CCC[SiH3] UIDUKLCLJMXFEO-UHFFFAOYSA-N 0.000 description 1
- 238000000746 purification Methods 0.000 description 1
- 239000008213 purified water Substances 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 238000012552 review Methods 0.000 description 1
- 239000000741 silica gel Substances 0.000 description 1
- 229910002027 silica gel Inorganic materials 0.000 description 1
- 238000006884 silylation reaction Methods 0.000 description 1
- 150000003385 sodium Chemical class 0.000 description 1
- 238000010532 solid phase synthesis reaction Methods 0.000 description 1
- 230000007480 spreading Effects 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 125000001424 substituent group Chemical group 0.000 description 1
- 239000000758 substrate Substances 0.000 description 1
- 239000000725 suspension Substances 0.000 description 1
- 230000008961 swelling Effects 0.000 description 1
- 230000002194 synthesizing effect Effects 0.000 description 1
- RYCLIXPGLDDLTM-UHFFFAOYSA-J tetrapotassium;phosphonato phosphate Chemical compound [K+].[K+].[K+].[K+].[O-]P([O-])(=O)OP([O-])([O-])=O RYCLIXPGLDDLTM-UHFFFAOYSA-J 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 238000013518 transcription Methods 0.000 description 1
- 230000035897 transcription Effects 0.000 description 1
- 238000012546 transfer Methods 0.000 description 1
- 230000009261 transgenic effect Effects 0.000 description 1
- 238000013519 translation Methods 0.000 description 1
- ZIBGPFATKBEMQZ-UHFFFAOYSA-N triethylene glycol Chemical group OCCOCCOCCO ZIBGPFATKBEMQZ-UHFFFAOYSA-N 0.000 description 1
- 238000010200 validation analysis Methods 0.000 description 1
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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
Landscapes
- 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)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662340947P | 2016-05-24 | 2016-05-24 | |
| US62/340,947 | 2016-05-24 | ||
| US201662429160P | 2016-12-02 | 2016-12-02 | |
| US62/429,160 | 2016-12-02 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| TW201805002A true TW201805002A (zh) | 2018-02-16 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW106117190A TW201805002A (zh) | 2016-05-24 | 2017-05-24 | 磷醯二胺嗎啉代寡聚物醫藥組合物 |
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)
| 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 |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| CA2906812A1 (en) * | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
-
2017
- 2017-05-24 US US16/302,484 patent/US20190275072A1/en not_active Abandoned
- 2017-05-24 TW TW106117190A patent/TW201805002A/zh unknown
- 2017-05-24 MX MX2018014129A patent/MX2018014129A/es unknown
- 2017-05-24 JP JP2018560674A patent/JP2019516730A/ja active Pending
- 2017-05-24 AU AU2017278699A patent/AU2017278699A1/en not_active Abandoned
- 2017-05-24 WO PCT/US2017/034265 patent/WO2017213854A1/en not_active Ceased
- 2017-05-24 SG SG11201809494VA patent/SG11201809494VA/en unknown
- 2017-05-24 CA CA3024178A patent/CA3024178A1/en not_active Abandoned
- 2017-05-24 KR KR1020187036635A patent/KR20190009343A/ko not_active Ceased
- 2017-05-24 MA MA045158A patent/MA45158A/fr unknown
- 2017-05-24 BR BR112018074299-6A patent/BR112018074299A2/pt not_active Application Discontinuation
- 2017-05-24 EP EP17731957.1A patent/EP3463390A1/en not_active Withdrawn
- 2017-05-24 CN CN201780030581.5A patent/CN109562123A/zh active Pending
-
2018
- 2018-11-15 IL IL263040A patent/IL263040A/en unknown
- 2018-12-19 CO CONC2018/0013828A patent/CO2018013828A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA3024178A1 (en) | 2017-12-14 |
| CN109562123A (zh) | 2019-04-02 |
| 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 |
| JP2019516730A (ja) | 2019-06-20 |
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