JP2018521993A5 - - Google Patents

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Publication number
JP2018521993A5
JP2018521993A5 JP2017566000A JP2017566000A JP2018521993A5 JP 2018521993 A5 JP2018521993 A5 JP 2018521993A5 JP 2017566000 A JP2017566000 A JP 2017566000A JP 2017566000 A JP2017566000 A JP 2017566000A JP 2018521993 A5 JP2018521993 A5 JP 2018521993A5
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Japan
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composition
day
effective amount
therapeutically effective
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JP2017566000A
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JP2018521993A (ja
JP6599484B2 (ja
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Priority claimed from PCT/US2016/038110 external-priority patent/WO2016205671A1/en
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JP2017566000A 2015-06-17 2016-06-17 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 Active JP6599484B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2019182192A Division JP7036782B2 (ja) 2015-06-17 2019-10-02 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
JP2019182191A Division JP6675037B2 (ja) 2015-06-17 2019-10-02 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

Publications (3)

Publication Number Publication Date
JP2018521993A JP2018521993A (ja) 2018-08-09
JP2018521993A5 true JP2018521993A5 (https=) 2019-07-04
JP6599484B2 JP6599484B2 (ja) 2019-10-30

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ID=57546446

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2017566000A Active JP6599484B2 (ja) 2015-06-17 2016-06-17 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
JP2019182191A Active JP6675037B2 (ja) 2015-06-17 2019-10-02 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
JP2019182192A Active JP7036782B2 (ja) 2015-06-17 2019-10-02 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

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JP2019182191A Active JP6675037B2 (ja) 2015-06-17 2019-10-02 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
JP2019182192A Active JP7036782B2 (ja) 2015-06-17 2019-10-02 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

Country Status (24)

Country Link
US (5) US10471087B2 (https=)
EP (3) EP3310362B1 (https=)
JP (3) JP6599484B2 (https=)
KR (3) KR20180039624A (https=)
CN (2) CN116726035A (https=)
AU (3) AU2016280293B2 (https=)
BR (2) BR112017027079B1 (https=)
CA (1) CA2989653A1 (https=)
CY (2) CY1122605T1 (https=)
DK (2) DK3505174T3 (https=)
ES (2) ES2748556T3 (https=)
HK (1) HK1252133A1 (https=)
HR (2) HRP20191794T1 (https=)
HU (2) HUE046399T2 (https=)
IL (3) IL275256B2 (https=)
LT (2) LT3310362T (https=)
MX (2) MX2017016425A (https=)
PL (2) PL3505174T3 (https=)
PT (2) PT3505174T (https=)
RS (2) RS59724B1 (https=)
RU (1) RU2721492C2 (https=)
SI (2) SI3505174T1 (https=)
SM (2) SMT202000399T1 (https=)
WO (1) WO2016205671A1 (https=)

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
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
WO2017112701A1 (en) 2015-12-22 2017-06-29 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
PT4293009T (pt) 2015-12-22 2025-09-08 Zogenix International Ltd Composições de fenfluramina e métodos de preparação das mesmas
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
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
US11628182B2 (en) * 2018-04-12 2023-04-18 Zogenix Mds, Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
KR20210009317A (ko) 2018-04-18 2021-01-26 더 트러스티스 오브 콜롬비아 유니버시티 인 더 시티 오브 뉴욕 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 초래된 질환에 대한 유전자 요법
EP3790537A1 (en) 2018-05-11 2021-03-17 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
AU2019384963B2 (en) 2018-11-19 2022-05-26 Zogenix International Limited Methods of treating Rett syndrome using fenfluramine
CA3150755A1 (en) * 2019-08-19 2021-02-25 Modis Therapeutics, Inc. Polymorphic forms of deoxycytidine, compositions comprising the same and uses
US20230000894A1 (en) * 2019-09-05 2023-01-05 Mitorainbow Therapeutics, Inc. Treating mitochondrial dna depletion disorders
AU2021236683A1 (en) 2020-03-19 2022-11-17 Intellia 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
EP4268829A4 (en) * 2020-12-28 2024-11-27 Yamasa Corporation MUSCLE ATROPHY INHIBITOR AND METHOD FOR INHIBITING MUSCLE ATROPHY
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
WO2024015453A1 (en) * 2022-07-12 2024-01-18 Zogenix Mds, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents
EP4687918A2 (en) * 2023-03-30 2026-02-11 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)

* Cited by examiner, † Cited by third party
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
IL144964A0 (en) * 1999-02-23 2002-06-30 Pro Neuron Inc Methods of treatment of mitochondrial disorders
CN100490818C (zh) * 2000-05-26 2009-05-27 诺瓦蒂斯有限公司 β-L-2′-脱氧核苷在制备用于治疗丁型肝炎病毒感染的药物中的用途
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
BR112017025839A2 (en) * 2015-06-05 2018-08-14 Fundació Hospital Universitari Vall D'hebron - Institut De Recerca treatment of mitochondrial diseases
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

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