CL2020002704A1 - Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial - Google Patents

Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial

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Publication number
CL2020002704A1
CL2020002704A1 CL2020002704A CL2020002704A CL2020002704A1 CL 2020002704 A1 CL2020002704 A1 CL 2020002704A1 CL 2020002704 A CL2020002704 A CL 2020002704A CL 2020002704 A CL2020002704 A CL 2020002704A CL 2020002704 A1 CL2020002704 A1 CL 2020002704A1
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CL
Chile
Prior art keywords
gene therapy
mitochondrial dna
treatment
dna depletion
involve
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CL2020002704A
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English (en)
Inventor
Michio Hirano
Hasan O Akman
Carlos Lopez-Gomez
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Univ Columbia
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Publication of CL2020002704A1 publication Critical patent/CL2020002704A1/es

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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
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Abstract

La invención se refiere en general, a un método de tratamiento para una enfermedad genética humana, tal como enfermedades caracterizadas por combinaciones desequilibradas de nucleótidos, por ejemplo, síndrome de agotamiento de ADN mitocondriales, y más específicamente, deficiencia de timidina cinasa 2 (TK2), usando terapia de gen. La terapia de gen puede involucrar la administración de uno o más constructos, tal como un vector viral, que contiene un ácido nucleico que codifica a una proteína funcional. La proteína funcional puede corresponder a un gen nuclear. Para tratamiento de deficiencia de TK2, la terapia de gen puede involucrar la administración de uno o más constructos, tal como un vector viral, que contiene un ácido nucleico que codifica a una enzima de proteína funcional TK2. El tratamiento puede involucrar también la administración de terapia farmacológica en conjunto con la terapia de gen. Los protocolos de tratamiento de la descripción, tal como aquellos involucran terapia de gen solos o en combinación con terapia farmacológica, pueden ser usados para tratar, prevenir, y/o curar varios otros trastornos de combinaciones desequilibradas de nucleósidos, especialmente aquellos encontrados en el síndrome de agotamiento de ADN mitocondrial.
CL2020002704A 2018-04-18 2020-10-19 Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial CL2020002704A1 (es)

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US201862659281P 2018-04-18 2018-04-18

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CL2020002704A1 true CL2020002704A1 (es) 2021-01-29

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US (1) US20210100917A1 (es)
EP (1) EP3781671A4 (es)
JP (1) JP2021522173A (es)
KR (1) KR20210009317A (es)
CN (1) CN112312929A (es)
AU (1) AU2019255745A1 (es)
BR (1) BR112020021324A2 (es)
CA (1) CA3097392A1 (es)
CL (1) CL2020002704A1 (es)
CO (1) CO2020014193A2 (es)
MX (1) MX2020010994A (es)
WO (1) WO2019204593A1 (es)

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US10351509B2 (en) 2015-12-22 2019-07-16 Zogenix International Limited Fenfluramine compositions and methods of preparing the same
US20170174614A1 (en) 2015-12-22 2017-06-22 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
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
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
EP3790537A1 (en) 2018-05-11 2021-03-17 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
WO2021102104A1 (en) * 2019-11-20 2021-05-27 University Of Massachusetts Aav-based delivery of thymine kinase 2
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
WO2022204555A1 (en) * 2021-03-26 2022-09-29 Zogenix Mds, Inc. Aqueous solutions containing purines and pyrimidines and uses thereof

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CA3097392A1 (en) 2019-10-24
CN112312929A (zh) 2021-02-02
EP3781671A4 (en) 2022-01-26
AU2019255745A1 (en) 2020-11-19
MX2020010994A (es) 2021-01-08
CO2020014193A2 (es) 2021-01-18
US20210100917A1 (en) 2021-04-08
JP2021522173A (ja) 2021-08-30
WO2019204593A1 (en) 2019-10-24
BR112020021324A2 (pt) 2021-01-19
EP3781671A1 (en) 2021-02-24
KR20210009317A (ko) 2021-01-26

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