MX2017016425A - Terapia con desoxinucleosidos para enfermedades generadas por grupos de nucleotidos desequilibrados, inclusive sindromes de agotamiento del adn mitocondrial. - Google Patents

Terapia con desoxinucleosidos para enfermedades generadas por grupos de nucleotidos desequilibrados, inclusive sindromes de agotamiento del adn mitocondrial.

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Publication number
MX2017016425A
MX2017016425A MX2017016425A MX2017016425A MX2017016425A MX 2017016425 A MX2017016425 A MX 2017016425A MX 2017016425 A MX2017016425 A MX 2017016425A MX 2017016425 A MX2017016425 A MX 2017016425A MX 2017016425 A MX2017016425 A MX 2017016425A
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MX
Mexico
Prior art keywords
mitochondrial dna
dna depletion
nucleotide pools
diseases caused
administration
Prior art date
Application number
MX2017016425A
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English (en)
Inventor
Hirano Michio
Garone Caterina
MARTI Ramon
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Univ Columbia
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Application filed by Univ Columbia filed Critical Univ Columbia
Publication of MX2017016425A publication Critical patent/MX2017016425A/es

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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/513Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
    • 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
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7068Compounds 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
    • 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
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7068Compounds 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
    • A61K31/7072Compounds 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
    • 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
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7076Compounds 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
    • 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
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7076Compounds 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
    • A61K31/708Compounds 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
    • AHUMAN NECESSITIES
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    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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Abstract

La invención se relaciona, en términos generales, con una terapia farmacológica para enfermedades genéticas humanas, específicamente aquellas caracterizadas por grupos de nucleótidos desequilibrados, más específicamente síndromes de agotamiento del ADN mitocondrial y más específicamente, la deficiencia de timidina cinasa 2 (TK2). La terapia farmacológica implica la administración de al menos un desoxinucleósido, o mezclas de estos. Para el tratamiento de la deficiencia de TK2, la terapia farmacológica implica la administración de desoxitimidina (dT) o desoxicitidina (dC) o mezclas de estas. Esta administración de desoxinucleósidos es aplicable a otros trastornos de grupos de nucleótidos desequilibrados, especialmente aquellos que se encuentran en el síndrome de agotamiento del ADN mitocondrial.
MX2017016425A 2015-06-17 2016-06-17 Terapia con desoxinucleosidos para enfermedades generadas por grupos de nucleotidos desequilibrados, inclusive sindromes de agotamiento del adn mitocondrial. MX2017016425A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17
PCT/US2016/038110 WO2016205671A1 (en) 2015-06-17 2016-06-17 Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes

Publications (1)

Publication Number Publication Date
MX2017016425A true MX2017016425A (es) 2018-12-11

Family

ID=57546446

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017016425A MX2017016425A (es) 2015-06-17 2016-06-17 Terapia con desoxinucleosidos para enfermedades generadas por grupos de nucleotidos desequilibrados, inclusive sindromes de agotamiento del adn mitocondrial.
MX2020000269A MX2020000269A (es) 2015-06-17 2016-06-17 Terapia con desoxinucleósidos para enfermedades generadas por grupos de nucleótidos desequilibrados, inclusive síndromes de agotamiento del adn mitocondrial.

Family Applications After (1)

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MX2020000269A MX2020000269A (es) 2015-06-17 2016-06-17 Terapia con desoxinucleósidos para enfermedades generadas por grupos de nucleótidos desequilibrados, inclusive síndromes de agotamiento del adn mitocondrial.

Country Status (23)

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US (4) US10471087B2 (es)
EP (3) EP3569236A1 (es)
JP (3) JP6599484B2 (es)
KR (2) KR20180039624A (es)
CN (2) CN116726035A (es)
AU (3) AU2016280293B2 (es)
BR (2) BR122020021913B1 (es)
CA (1) CA2989653A1 (es)
CY (2) CY1122605T1 (es)
DK (2) DK3505174T3 (es)
ES (2) ES2808148T3 (es)
HK (1) HK1252133A1 (es)
HR (2) HRP20191794T1 (es)
HU (2) HUE046399T2 (es)
IL (3) IL275256B2 (es)
LT (2) LT3310362T (es)
MX (2) MX2017016425A (es)
PL (2) PL3310362T3 (es)
PT (2) PT3505174T (es)
RS (2) RS59724B1 (es)
RU (2) RU2721492C2 (es)
SI (2) SI3310362T1 (es)
WO (1) WO2016205671A1 (es)

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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
RU2721492C2 (ru) * 2015-06-17 2020-05-19 Зе Трастис Оф Коламбия Юниверсити Ин Зе Сити Оф Нью-Йорк Дезоксинуклеозидная терапия заболеваний, вызванных несбалансированными пулами нуклеотидов, в том числе синдромов истощения митохондриальной днк
JP6893213B2 (ja) 2015-12-22 2021-06-23 ゾゲニクス インターナショナル リミテッド フェンフルラミン組成物およびその調製法
JP2019507111A (ja) 2015-12-22 2019-03-14 ゾゲニクス インターナショナル リミテッド 代謝抵抗性フェンフルラミン類縁体およびその使用法
CA3032996A1 (en) 2016-08-24 2018-03-01 Zogenix International Limited Formulation for inhibiting formation of 5-ht 2b agonists and methods of using same
US11649442B2 (en) 2017-09-08 2023-05-16 The Regents Of The University Of California RNA-guided endonuclease fusion polypeptides and methods of use thereof
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
BR112020020804A2 (pt) * 2018-04-12 2021-01-12 Modis Therapeutics Inc. Pró-fármacos de desoxinucleosídeos para tratamento de doenças causadas por agrupamentos de nucleotídeos não balanceados
EP3790537A1 (en) 2018-05-11 2021-03-17 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
AU2020334060A1 (en) * 2019-08-19 2022-03-24 Zogenix, Inc. Polymorphic forms of deoxycytidine, compositions comprising the same and uses
CA3146835A1 (en) * 2019-09-05 2021-03-11 Mitorainbow Therapeutics, Inc. Treating mitochondrial dna depletion disorders
WO2021188840A1 (en) 2020-03-19 2021-09-23 Rewrite Therapeutics, Inc. Methods and compositions for directed genome editing
US11612574B2 (en) 2020-07-17 2023-03-28 Zogenix International Limited Method of treating patients infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)
US20220096514A1 (en) * 2020-09-29 2022-03-31 Zogenix International Limited Methods of treating thymidine kinase 2 deficiency by administering deoxycytidine and deoxythymidine
EP4268829A1 (en) * 2020-12-28 2023-11-01 Yamasa Corporation Muscular atrophy inhibitor and method for inhibiting muscular atrophy
WO2022204555A1 (en) * 2021-03-26 2022-09-29 Zogenix Mds, Inc. Aqueous solutions containing purines and pyrimidines and uses thereof
WO2022266237A1 (en) * 2021-06-18 2022-12-22 Zogenix Mds, Inc. Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools
US20240016938A1 (en) * 2022-07-12 2024-01-18 Zogenix Mds, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents

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RU2300381C2 (ru) * 1998-08-10 2007-06-10 Айденикс (Кайман) Лимитед β-L-2'-ДЕЗОКСИНУКЛЕОЗИДЫ ДЛЯ ЛЕЧЕНИЯ ГЕПАТИТА В
KR100879559B1 (ko) * 1999-02-23 2009-01-22 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 미토콘드리아 장애의 치료 또는 예방용 제약조성물
ES2227203T3 (es) * 2000-05-26 2005-04-01 Idenix (Cayman) Limited Metodos para tratar la infeccion por el virus de la hepatitis delta con beta-1-2'-desoxinucleosidos.
WO2012125848A2 (en) * 2011-03-16 2012-09-20 Baylor College Of Medicine A method for comprehensive sequence analysis using deep sequencing technology
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
US11337980B2 (en) * 2015-06-05 2022-05-24 Fundació Hospital Universitari Vall D'hebron-Institut De Recerca Treatment of mitochondrial diseases
RU2721492C2 (ru) * 2015-06-17 2020-05-19 Зе Трастис Оф Коламбия Юниверсити Ин Зе Сити Оф Нью-Йорк Дезоксинуклеозидная терапия заболеваний, вызванных несбалансированными пулами нуклеотидов, в том числе синдромов истощения митохондриальной днк

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AU2020204042B2 (en) 2021-07-15
US10471087B2 (en) 2019-11-12
CN116726035A (zh) 2023-09-12
US11110111B2 (en) 2021-09-07
EP3310362A1 (en) 2018-04-25
KR20220018623A (ko) 2022-02-15
HRP20191794T1 (hr) 2020-02-07
ES2748556T3 (es) 2020-03-17
MX2020000269A (es) 2022-01-03
JP2018521993A (ja) 2018-08-09
PL3310362T3 (pl) 2020-04-30
RU2018101305A3 (es) 2019-10-31
AU2021250841A1 (en) 2021-11-04
DK3310362T3 (da) 2019-10-07
CY1122605T1 (el) 2021-03-12
WO2016205671A9 (en) 2018-01-04
US20180133241A1 (en) 2018-05-17
EP3310362A4 (en) 2018-12-19
RS59724B1 (sr) 2020-01-31
EP3310362B1 (en) 2019-08-28
HK1252133A1 (zh) 2019-05-17
JP6599484B2 (ja) 2019-10-30
IL275255B (en) 2021-04-29
AU2021250841B2 (en) 2023-02-09
RU2020112901A (ru) 2020-06-05
ES2808148T3 (es) 2021-02-25
HUE050678T2 (hu) 2020-12-28
LT3310362T (lt) 2019-11-11
AU2020204042A1 (en) 2020-07-09
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
PT3310362T (pt) 2019-10-29
US20230277577A1 (en) 2023-09-07
IL275255A (en) 2020-07-30
JP2020019792A (ja) 2020-02-06
JP6675037B2 (ja) 2020-04-01
IL275256B2 (en) 2023-09-01
AU2016280293B2 (en) 2020-07-02
CN107847512A (zh) 2018-03-27
CY1123107T1 (el) 2021-10-29
KR20180039624A (ko) 2018-04-18
LT3505174T (lt) 2020-09-10
HUE046399T2 (hu) 2020-03-30
SI3310362T1 (sl) 2020-02-28
IL256331A (en) 2018-02-28
HRP20200949T1 (hr) 2020-10-02
IL275256A (en) 2020-07-30
BR112017027079B1 (pt) 2021-04-27
SI3505174T1 (sl) 2021-02-26
AU2016280293A1 (en) 2018-02-08
EP3505174B1 (en) 2020-04-29
PL3505174T3 (pl) 2020-08-24
DK3505174T3 (da) 2020-07-13
JP2020019791A (ja) 2020-02-06
RS60572B1 (sr) 2020-08-31
RU2018101305A (ru) 2019-07-17
US11666592B2 (en) 2023-06-06
RU2721492C2 (ru) 2020-05-19
WO2016205671A1 (en) 2016-12-22
JP7036782B2 (ja) 2022-03-15
US20200038424A1 (en) 2020-02-06
BR122020021913B1 (pt) 2021-07-06
EP3505174A1 (en) 2019-07-03
IL275256B1 (en) 2023-05-01
CA2989653A1 (en) 2016-12-22
IL256331B (en) 2020-08-31
EP3569236A1 (en) 2019-11-20

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