CO2020014193A2 - Terapia genética para las enfermedades causadas por piscinas de nucleótidos desequilibradas, incluyendo los síndromes de depleción del adn mitocondrial - Google Patents

Terapia genética para las enfermedades causadas por piscinas de nucleótidos desequilibradas, incluyendo los síndromes de depleción del adn mitocondrial

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Publication number
CO2020014193A2
CO2020014193A2 CONC2020/0014193A CO2020014193A CO2020014193A2 CO 2020014193 A2 CO2020014193 A2 CO 2020014193A2 CO 2020014193 A CO2020014193 A CO 2020014193A CO 2020014193 A2 CO2020014193 A2 CO 2020014193A2
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Colombia
Prior art keywords
gene therapy
mitochondrial dna
treatment
dna depletion
involve
Prior art date
Application number
CONC2020/0014193A
Other languages
English (en)
Spanish (es)
Inventor
Michio Hirano
Hasan O Akman
Carlos Lopez-Gomez
Original Assignee
Univ Columbia
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ Columbia filed Critical Univ Columbia
Publication of CO2020014193A2 publication Critical patent/CO2020014193A2/es

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    • A—HUMAN NECESSITIES
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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
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/45—Transferases (2)
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    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/10—Transferases (2.)
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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
    • C12N9/1211—Thymidine kinase (2.7.1.21)
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • C12Y117/00—Oxidoreductases acting on CH or CH2 groups (1.17)
    • C12Y117/04—Oxidoreductases acting on CH or CH2 groups (1.17) with a disulfide as acceptor (1.17.3)
    • C12Y117/04001—Ribonucleoside-diphosphate reductase (1.17.4.1)
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    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02004—Thymidine phosphorylase (2.4.2.4)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01021—Thymidine kinase (2.7.1.21)
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01076—Deoxyadenosine kinase (2.7.1.76)
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    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01113—Deoxyguanosine kinase (2.7.1.113)
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
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CONC2020/0014193A 2018-04-18 2020-11-17 Terapia genética para las enfermedades causadas por piscinas de nucleótidos desequilibradas, incluyendo los síndromes de depleción del adn mitocondrial CO2020014193A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862659281P 2018-04-18 2018-04-18
PCT/US2019/028108 WO2019204593A1 (en) 2018-04-18 2019-04-18 Gene therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes

Publications (1)

Publication Number Publication Date
CO2020014193A2 true CO2020014193A2 (es) 2021-01-18

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CONC2020/0014193A CO2020014193A2 (es) 2018-04-18 2020-11-17 Terapia genética para las enfermedades causadas por piscinas de nucleótidos desequilibradas, incluyendo los síndromes de depleción del adn mitocondrial

Country Status (13)

Country Link
US (1) US12419970B2 (https=)
EP (1) EP3781671A4 (https=)
JP (1) JP7769355B2 (https=)
KR (1) KR20210009317A (https=)
CN (1) CN112312929A (https=)
AU (1) AU2019255745A1 (https=)
BR (1) BR112020021324A2 (https=)
CA (1) CA3097392A1 (https=)
CL (1) CL2020002704A1 (https=)
CO (1) CO2020014193A2 (https=)
IL (1) IL278130B1 (https=)
MX (1) MX2020010994A (https=)
WO (1) WO2019204593A1 (https=)

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AU2017315273B2 (en) 2016-08-24 2021-04-29 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
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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
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KR20250004463A (ko) * 2023-06-30 2025-01-08 사회복지법인 삼성생명공익재단 Hydra NDi1가 탑재된 재조합 아데노부속바이러스 벡터를 포함하는, 미토콘드리아 복합체 1 이상질환의 예방, 개선 또는 치료용 조성물
CN120060299B (zh) * 2025-02-21 2025-11-28 三峡大学 一种密码子优化的twnk基因、腺相关病毒载体及其应用

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JP2021522173A (ja) 2021-08-30
US12419970B2 (en) 2025-09-23
EP3781671A1 (en) 2021-02-24
MX2020010994A (es) 2021-01-08
EP3781671A4 (en) 2022-01-26
AU2019255745A1 (en) 2020-11-19
IL278130B1 (en) 2026-01-01
JP7769355B2 (ja) 2025-11-13
US20210100917A1 (en) 2021-04-08
IL278130A (en) 2020-12-31
KR20210009317A (ko) 2021-01-26
CL2020002704A1 (es) 2021-01-29
CA3097392A1 (en) 2019-10-24
WO2019204593A1 (en) 2019-10-24
CN112312929A (zh) 2021-02-02

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