CA3096264A1 - Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools - Google Patents
Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools Download PDFInfo
- Publication number
- CA3096264A1 CA3096264A1 CA3096264A CA3096264A CA3096264A1 CA 3096264 A1 CA3096264 A1 CA 3096264A1 CA 3096264 A CA3096264 A CA 3096264A CA 3096264 A CA3096264 A CA 3096264A CA 3096264 A1 CA3096264 A1 CA 3096264A1
- Authority
- CA
- Canada
- Prior art keywords
- optionally substituted
- alkyl
- base
- prodrug
- formula
- 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
Links
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
- A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
- A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
- A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
- A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
- A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
- A61K31/4439—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
- A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
-
- 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
- A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
- A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
- A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
- A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
- A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
- A61K31/7072—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
-
- 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
- A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
- A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
- A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
- A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
- A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
- A61K31/708—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid having oxo groups directly attached to the purine ring system, e.g. guanosine, guanylic acid
-
- 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
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
- C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/04—Ortho-condensed systems
-
- 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
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Nutrition Science (AREA)
- Physiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3096264A1 true CA3096264A1 (en) | 2019-10-17 |
Family
ID=68163825
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3096264A Pending CA3096264A1 (en) | 2018-04-12 | 2019-04-12 | Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools |
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
Also Published As
| Publication number | Publication date |
|---|---|
| EP3773603A1 (en) | 2021-02-17 |
| IL277725B2 (en) | 2024-02-01 |
| IL277725A (en) | 2020-11-30 |
| US20230181617A1 (en) | 2023-06-15 |
| WO2019200340A1 (en) | 2019-10-17 |
| JP7784411B2 (ja) | 2025-12-11 |
| US12419901B2 (en) | 2025-09-23 |
| US20210077519A1 (en) | 2021-03-18 |
| JP2021521210A (ja) | 2021-08-26 |
| JP2024038011A (ja) | 2024-03-19 |
| US11628182B2 (en) | 2023-04-18 |
| CN112384228A (zh) | 2021-02-19 |
| AU2019253023A1 (en) | 2020-11-12 |
| BR112020020804A2 (pt) | 2021-01-12 |
| MX2020010667A (es) | 2021-01-08 |
| JP7620781B2 (ja) | 2025-01-24 |
| IL277725B1 (en) | 2023-10-01 |
| US20230181616A1 (en) | 2023-06-15 |
| EP3773603A4 (en) | 2022-03-30 |
| AU2019253023B2 (en) | 2025-01-23 |
| AU2025201857A1 (en) | 2025-04-03 |
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| US12419901B2 (en) | Prodrugs of deoxynucleosides for treatment of diseases caused by unbalanced nucleotide pools | |
| US11633418B2 (en) | Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome | |
| US20250049836A1 (en) | Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools | |
| RU2827432C2 (ru) | Дезоксинуклеозидная терапия заболеваний, вызванных несбалансированными пулами нуклеотидов, в том числе синдромов истощения митохондриальной днк | |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20231228 |
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| R11 | Change to the name of applicant or owner or transfer of ownership requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-R10-R11-R127 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: TRANSFER RECORDAL REQUEST OR RESPONSE Effective date: 20240906 |
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| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20240906 |
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| R14 | Transfer of ownership recorded |
Free format text: ST27 STATUS EVENT CODE: A-2-2-R10-R14-R129 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: TRANSFER REQUIREMENTS DETERMINED COMPLIANT Effective date: 20240911 |
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| D15 | Examination report completed |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT Effective date: 20250307 |
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| MFA | Maintenance fee for application paid |
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| U00 | Fee paid |
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| W00 | Other event occurred |
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| P13 | Application amended |
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| MFA | Maintenance fee for application paid |
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| U00 | Fee paid |
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| U11 | Full renewal or maintenance fee paid |
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