BR112017027079B1 - Uso de uma composição compreendendo desoxinucleosídeos para o tratamento de síndrome de depleção do dna mitocondrial relacionada a timidina quinase - Google Patents

Uso de uma composição compreendendo desoxinucleosídeos para o tratamento de síndrome de depleção do dna mitocondrial relacionada a timidina quinase Download PDF

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Publication number
BR112017027079B1
BR112017027079B1 BR112017027079-0A BR112017027079A BR112017027079B1 BR 112017027079 B1 BR112017027079 B1 BR 112017027079B1 BR 112017027079 A BR112017027079 A BR 112017027079A BR 112017027079 B1 BR112017027079 B1 BR 112017027079B1
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Brazil
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day
administration
mitochondrial dna
deficiency
mitochondrial
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BR112017027079-0A
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English (en)
Portuguese (pt)
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Michio Hirano
Caterina Garone
Ramon Marti
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The Trustees Of Columbia University In The City Of New York
Fundacio Hospital Universitari Vall D'hebron-Institut De Recerca
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Priority to BR122020021913-0A priority Critical patent/BR122020021913B1/pt
Publication of BR112017027079B1 publication Critical patent/BR112017027079B1/pt

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    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/513—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
    • A61K31/7076—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 containing purines, e.g. adenosine, adenylic 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
    • A61K31/7076—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 containing purines, e.g. adenosine, adenylic acid
    • A61K31/708—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 containing purines, e.g. adenosine, adenylic acid having oxo groups directly attached to the purine ring system, e.g. guanosine, guanylic acid
    • A—HUMAN NECESSITIES
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    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12Q1/6813—Hybridisation assays
    • C12Q1/6827—Hybridisation assays for detection of mutation or polymorphism
    • G—PHYSICS
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    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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BR112017027079-0A 2015-06-17 2016-06-17 Uso de uma composição compreendendo desoxinucleosídeos para o tratamento de síndrome de depleção do dna mitocondrial relacionada a timidina quinase BR112017027079B1 (pt)

Priority Applications (1)

Application Number Priority Date Filing Date Title
BR122020021913-0A BR122020021913B1 (pt) 2015-06-17 2016-06-17 Uso de uma composição compreendendo desoxinucleosídeos para o tratamento de síndrome de depleção do dna mitocondrial (mdds) em um sujeito humano

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

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BR112017027079B1 true BR112017027079B1 (pt) 2021-04-27

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BR112017027079-0A BR112017027079B1 (pt) 2015-06-17 2016-06-17 Uso de uma composição compreendendo desoxinucleosídeos para o tratamento de síndrome de depleção do dna mitocondrial relacionada a timidina quinase
BR122020021913-0A BR122020021913B1 (pt) 2015-06-17 2016-06-17 Uso de uma composição compreendendo desoxinucleosídeos para o tratamento de síndrome de depleção do dna mitocondrial (mdds) em um sujeito humano

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BR122020021913-0A BR122020021913B1 (pt) 2015-06-17 2016-06-17 Uso de uma composição compreendendo desoxinucleosídeos para o tratamento de síndrome de depleção do dna mitocondrial (mdds) em um sujeito humano

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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=)
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LT (2) LT3310362T (https=)
MX (2) MX2017016425A (https=)
PL (2) PL3505174T3 (https=)
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RS (2) RS59724B1 (https=)
RU (1) RU2721492C2 (https=)
SI (2) SI3505174T1 (https=)
SM (2) SMT202000399T1 (https=)
WO (1) WO2016205671A1 (https=)

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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
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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
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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
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

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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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SMT202000399T1 (it) 2020-09-10
RU2020112901A (ru) 2020-06-05
SI3505174T1 (sl) 2021-02-26
HK1252133A1 (zh) 2019-05-17
US20250360154A1 (en) 2025-11-27
AU2016280293B2 (en) 2020-07-02
PL3310362T3 (pl) 2020-04-30
CN116726035A (zh) 2023-09-12
EP3505174B1 (en) 2020-04-29
MX2017016425A (es) 2018-12-11
SMT201900571T1 (it) 2019-11-13
EP3505174A1 (en) 2019-07-03
KR20220018623A (ko) 2022-02-15
IL256331B (en) 2020-08-31
HUE046399T2 (hu) 2020-03-30
EP3569236A1 (en) 2019-11-20
AU2020204042B2 (en) 2021-07-15
US11110111B2 (en) 2021-09-07
US20180133241A1 (en) 2018-05-17
JP2018521993A (ja) 2018-08-09
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AU2020204042A1 (en) 2020-07-09
SI3310362T1 (sl) 2020-02-28
DK3310362T3 (da) 2019-10-07
US12251392B2 (en) 2025-03-18
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US11666592B2 (en) 2023-06-06
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