KR20180039624A - 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법 - Google Patents

미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법 Download PDF

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KR20180039624A
KR20180039624A KR1020187001496A KR20187001496A KR20180039624A KR 20180039624 A KR20180039624 A KR 20180039624A KR 1020187001496 A KR1020187001496 A KR 1020187001496A KR 20187001496 A KR20187001496 A KR 20187001496A KR 20180039624 A KR20180039624 A KR 20180039624A
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day
disease
composition
nucleotide pool
subject
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미치오 히라노
케이트리나 가론
라몬 마르띠
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더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕
펀다시오 호스피탈 유니버시타리 발 드헤브론-인스티튜트 디 레세르카
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Priority to KR1020227002790A priority Critical patent/KR20220018623A/ko
Publication of KR20180039624A publication Critical patent/KR20180039624A/ko
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KR1020187001496A 2015-06-17 2016-06-17 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법 Ceased KR20180039624A (ko)

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KR1020227002790A KR20220018623A (ko) 2015-06-17 2016-06-17 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법

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US201562180914P 2015-06-17 2015-06-17
US62/180,914 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

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KR1020187001496A Ceased KR20180039624A (ko) 2015-06-17 2016-06-17 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법
KR1020257009842A Pending KR20250048602A (ko) 2015-06-17 2016-06-17 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법
KR1020227002790A Ceased KR20220018623A (ko) 2015-06-17 2016-06-17 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법

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KR1020257009842A Pending KR20250048602A (ko) 2015-06-17 2016-06-17 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법
KR1020227002790A Ceased KR20220018623A (ko) 2015-06-17 2016-06-17 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 야기된 질환에 대한 데옥시뉴클레오타이드 요법

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SI (2) SI3310362T1 (enExample)
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Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9549909B2 (en) 2013-05-03 2017-01-24 The Katholieke Universiteit Leuven Method for the treatment of dravet syndrome
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
US10471087B2 (en) * 2015-06-17 2019-11-12 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes
EP3393470B1 (en) 2015-12-22 2021-01-20 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
CA3007286A1 (en) 2015-12-22 2017-06-29 Zogenix International Limited Fenfluramine compositions and methods of preparing the same
MX2019001799A (es) 2016-08-24 2019-06-13 Zogenix International Ltd Formulacion para inhibir la formacion de agonistas de 5-ht2b y metodos de utilizacion de la misma.
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
WO2019200340A1 (en) 2018-04-12 2019-10-17 Modis Therapeutics Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
MX2020010994A (es) 2018-04-18 2021-01-08 Univ Columbia Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial.
WO2019216919A1 (en) 2018-05-11 2019-11-14 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
EA202191079A1 (ru) 2018-11-19 2021-09-20 Зодженикс Интернэшнл Лимитед Способы лечения синдрома ретта с применением фенфлурамина
EP4017502A4 (en) * 2019-08-19 2023-11-01 Modis Therapeutics Inc. POLYMORPH FORMS OF DEOXYCYTIDINE, COMPOSITIONS COMPRISING THEM AND USES
CN114340641B (zh) * 2019-09-05 2025-04-04 米托瑞因博治疗股份有限公司 治疗线粒体dna缺失障碍
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
WO2022145439A1 (ja) * 2020-12-28 2022-07-07 ヤマサ醤油株式会社 筋萎縮抑制剤及び筋萎縮を抑制させる方法
CN117120060A (zh) * 2021-03-26 2023-11-24 周格尼克斯-莫迪斯公司 含有嘌呤和嘧啶的水溶液及其用途
EP4355336A4 (en) * 2021-06-18 2025-05-07 UCB Biosciences, Inc. DEOXYNUCLEOSIDE PRODRUGS FOR THE TREATMENT OF MITOCHONDRIAL DISEASES CAUSED BY UNBALANCED NUCLEOTIDE GROUPS
EP4554596A1 (en) * 2022-07-12 2025-05-21 UCB Biosciences, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents
WO2024206595A2 (en) * 2023-03-30 2024-10-03 The Trustees Of Columbia University In The City Of New York Pharmacological therapy for mitochondrial dna depletion deletions syndrome involving mutations in the guk1 gene

Family Cites Families (7)

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Publication number Priority date Publication date Assignee Title
CN1911237B (zh) * 1998-08-10 2010-09-15 艾丹尼克斯(开曼)有限公司 用于治疗乙型肝炎的β-L-2＇-脱氧-核苷
KR100879559B1 (ko) * 1999-02-23 2009-01-22 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 미토콘드리아 장애의 치료 또는 예방용 제약조성물
US6596700B2 (en) * 2000-05-26 2003-07-22 Idenix Pharmaceuticals Inc. Methods of treating hepatitis delta virus infection with β-L-2'-deoxy-nucleosides
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
PL3302499T3 (pl) * 2015-06-05 2023-07-24 Fundació Hospital Universitari Vall D'hebron - Institut De Recerca Leczenie chorób mitochondrialnych
US10471087B2 (en) * 2015-06-17 2019-11-12 The Trustees Of Columbia University In The City Of New York Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes

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