JPWO2019200340A5 - - Google Patents
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- Publication number
- JPWO2019200340A5 JPWO2019200340A5 JP2020556284A JP2020556284A JPWO2019200340A5 JP WO2019200340 A5 JPWO2019200340 A5 JP WO2019200340A5 JP 2020556284 A JP2020556284 A JP 2020556284A JP 2020556284 A JP2020556284 A JP 2020556284A JP WO2019200340 A5 JPWO2019200340 A5 JP WO2019200340A5
- Authority
- JP
- Japan
- Prior art keywords
- optionally substituted
- pharmaceutical composition
- alkyl
- optional
- prodrug
- 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.)
- Granted
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- 239000008194 pharmaceutical composition Substances 0.000 claims 20
- 125000000217 alkyl group Chemical group 0.000 claims 15
- 229940002612 prodrug Drugs 0.000 claims 15
- 239000000651 prodrug Substances 0.000 claims 15
- 125000003107 substituted aryl group Chemical group 0.000 claims 9
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 8
- 125000000623 heterocyclic group Chemical group 0.000 claims 8
- 229910052739 hydrogen Inorganic materials 0.000 claims 7
- 239000001257 hydrogen Substances 0.000 claims 7
- 125000004435 hydrogen atom Chemical class [H]* 0.000 claims 7
- 125000003342 alkenyl group Chemical group 0.000 claims 5
- 125000000304 alkynyl group Chemical group 0.000 claims 5
- 150000001413 amino acids Chemical class 0.000 claims 5
- 230000007812 deficiency Effects 0.000 claims 5
- 125000005913 (C3-C6) cycloalkyl group Chemical group 0.000 claims 4
- 125000003277 amino group Chemical group 0.000 claims 4
- 150000001875 compounds Chemical class 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 4
- 208000035475 disorder Diseases 0.000 claims 4
- -1 amino acid esters Chemical class 0.000 claims 3
- 125000000392 cycloalkenyl group Chemical group 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 125000002252 acyl group Chemical group 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 229940079593 drug Drugs 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 125000001072 heteroaryl group Chemical group 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 102100036853 Deoxyguanosine kinase, mitochondrial Human genes 0.000 claims 1
- 101000928003 Homo sapiens Deoxyguanosine kinase, mitochondrial Proteins 0.000 claims 1
- 101000969776 Homo sapiens Protein Mpv17 Proteins 0.000 claims 1
- 101001095783 Homo sapiens Ribonucleoside-diphosphate reductase subunit M2 B Proteins 0.000 claims 1
- 101000661446 Homo sapiens Succinate-CoA ligase [ADP-forming] subunit beta, mitochondrial Proteins 0.000 claims 1
- 101000832009 Homo sapiens Succinate-CoA ligase [ADP/GDP-forming] subunit alpha, mitochondrial Proteins 0.000 claims 1
- 101000796134 Homo sapiens Thymidine phosphorylase Proteins 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-N Phosphoric acid Chemical class OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 claims 1
- 102100021273 Protein Mpv17 Human genes 0.000 claims 1
- 102100038013 Ribonucleoside-diphosphate reductase subunit M2 B Human genes 0.000 claims 1
- 102100037811 Succinate-CoA ligase [ADP-forming] subunit beta, mitochondrial Human genes 0.000 claims 1
- 102100024241 Succinate-CoA ligase [ADP/GDP-forming] subunit alpha, mitochondrial Human genes 0.000 claims 1
- 102100031372 Thymidine phosphorylase Human genes 0.000 claims 1
- 125000003545 alkoxy group Chemical group 0.000 claims 1
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims 1
- 125000004390 alkyl sulfonyl group Chemical group 0.000 claims 1
- 125000005140 aralkylsulfonyl group Chemical group 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 125000004391 aryl sulfonyl group Chemical group 0.000 claims 1
- 150000001720 carbohydrates Chemical class 0.000 claims 1
- 235000014633 carbohydrates Nutrition 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 125000004122 cyclic group Chemical group 0.000 claims 1
- 125000000753 cycloalkyl group Chemical group 0.000 claims 1
- 239000001177 diphosphate Substances 0.000 claims 1
- XPPKVPWEQAFLFU-UHFFFAOYSA-J diphosphate(4-) Chemical class [O-]P([O-])(=O)OP([O-])([O-])=O XPPKVPWEQAFLFU-UHFFFAOYSA-J 0.000 claims 1
- 235000011180 diphosphates Nutrition 0.000 claims 1
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 150000004712 monophosphates Chemical class 0.000 claims 1
- 150000003904 phospholipids Chemical class 0.000 claims 1
- 150000003013 phosphoric acid derivatives Chemical class 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 150000003459 sulfonic acid esters Chemical class 0.000 claims 1
- 239000001226 triphosphate Substances 0.000 claims 1
- 235000011178 triphosphate Nutrition 0.000 claims 1
- 125000002264 triphosphate group Chemical class [H]OP(=O)(O[H])OP(=O)(O[H])OP(=O)(O[H])O* 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023215758A JP7784411B2 (ja) | 2018-04-12 | 2023-12-21 | 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862656861P | 2018-04-12 | 2018-04-12 | |
| US62/656,861 | 2018-04-12 | ||
| PCT/US2019/027364 WO2019200340A1 (en) | 2018-04-12 | 2019-04-12 | Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023215758A Division JP7784411B2 (ja) | 2018-04-12 | 2023-12-21 | 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021521210A JP2021521210A (ja) | 2021-08-26 |
| JP2021521210A5 JP2021521210A5 (https=) | 2022-04-19 |
| JPWO2019200340A5 true JPWO2019200340A5 (https=) | 2022-04-19 |
| JP7620781B2 JP7620781B2 (ja) | 2025-01-24 |
Family
ID=68163825
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020556284A Active JP7620781B2 (ja) | 2018-04-12 | 2019-04-12 | 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ |
| JP2023215758A Active JP7784411B2 (ja) | 2018-04-12 | 2023-12-21 | 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023215758A Active JP7784411B2 (ja) | 2018-04-12 | 2023-12-21 | 不均衡なヌクレオチドプールによって引き起こされた疾患を治療するためのデオキシヌクレオシドのプロドラッグ |
Country Status (10)
| Country | Link |
|---|---|
| US (3) | US11628182B2 (https=) |
| EP (1) | EP3773603A4 (https=) |
| JP (2) | JP7620781B2 (https=) |
| CN (1) | CN112384228A (https=) |
| AU (2) | AU2019253023B2 (https=) |
| BR (1) | BR112020020804A2 (https=) |
| CA (1) | CA3096264A1 (https=) |
| IL (1) | IL277725B2 (https=) |
| MX (1) | MX2020010667A (https=) |
| WO (1) | WO2019200340A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11628182B2 (en) | 2018-04-12 | 2023-04-18 | Zogenix Mds, Inc. | Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools |
| EP4313068A4 (en) * | 2021-03-26 | 2024-12-25 | UCB Biosciences, Inc. | Aqueous solutions containing purines and pyrimidines and uses thereof |
| US20230000890A1 (en) * | 2021-06-18 | 2023-01-05 | Zogenix Mds, Inc. | Prodrugs of Deoxynucleosides for Treatment of Mitochondrial Diseases Caused by Unbalanced Nucleotide Pools |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| EP0753525A4 (en) | 1994-03-31 | 1997-09-03 | Nippon Shinyaku Co Ltd | CYCLIC NUCLEOTIDE DERIVATIVES |
| US6875751B2 (en) | 2000-06-15 | 2005-04-05 | Idenix Pharmaceuticals, Inc. | 3′-prodrugs of 2′-deoxy-β-L-nucleosides |
| MY141594A (en) | 2000-06-15 | 2010-05-14 | Novirio Pharmaceuticals Ltd | 3'-PRODRUGS OF 2'-DEOXY-ß-L-NUCLEOSIDES |
| WO2002057425A2 (en) | 2001-01-22 | 2002-07-25 | Merck & Co., Inc. | Nucleoside derivatives as inhibitors of rna-dependent rna viral polymerase |
| US10292996B2 (en) | 2015-03-26 | 2019-05-21 | The Trustees Of Columbia University In The City Of New York | Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome |
| JP6599484B2 (ja) | 2015-06-17 | 2019-10-30 | ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク | ミトコンドリアｄｎａ枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法 |
| US10745435B2 (en) * | 2015-11-16 | 2020-08-18 | Cerecor, Inc. | Nucleic acid prodrugs |
| BR112018076913A2 (pt) | 2016-06-24 | 2019-04-02 | Emory University | fosforamidatos para o tratamento do vírus da hepatite b |
| US11628182B2 (en) | 2018-04-12 | 2023-04-18 | Zogenix Mds, Inc. | Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools |
-
2019
- 2019-04-12 US US17/046,865 patent/US11628182B2/en active Active
- 2019-04-12 JP JP2020556284A patent/JP7620781B2/ja active Active
- 2019-04-12 AU AU2019253023A patent/AU2019253023B2/en active Active
- 2019-04-12 MX MX2020010667A patent/MX2020010667A/es unknown
- 2019-04-12 EP EP19784627.2A patent/EP3773603A4/en active Pending
- 2019-04-12 BR BR112020020804-3A patent/BR112020020804A2/pt unknown
- 2019-04-12 CA CA3096264A patent/CA3096264A1/en active Pending
- 2019-04-12 IL IL277725A patent/IL277725B2/en unknown
- 2019-04-12 WO PCT/US2019/027364 patent/WO2019200340A1/en not_active Ceased
- 2019-04-12 CN CN201980038898.2A patent/CN112384228A/zh active Pending
-
2023
- 2023-02-10 US US18/167,317 patent/US20230181617A1/en active Pending
- 2023-02-10 US US18/167,308 patent/US12419901B2/en active Active
- 2023-12-21 JP JP2023215758A patent/JP7784411B2/ja active Active
-
2025
- 2025-03-14 AU AU2025201857A patent/AU2025201857A1/en active Pending
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