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
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    • 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
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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
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    • 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
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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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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

Publications (1)

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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) EP3505174B1 (https=)
JP (3) JP6599484B2 (https=)
KR (3) KR20250048602A (https=)
CN (2) CN107847512A (https=)
AU (3) AU2016280293B2 (https=)
BR (2) BR112017027079B1 (https=)
CA (1) CA2989653A1 (https=)
CY (2) CY1122605T1 (https=)
DK (2) DK3310362T3 (https=)
ES (2) ES2748556T3 (https=)
HK (1) HK1252133A1 (https=)
HR (2) HRP20191794T1 (https=)
HU (2) HUE046399T2 (https=)
IL (3) IL275256B2 (https=)
LT (2) LT3505174T (https=)
MX (2) MX2017016425A (https=)
PL (2) PL3505174T3 (https=)
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RS (2) RS60572B1 (https=)
RU (1) RU2721492C2 (https=)
SI (2) SI3310362T1 (https=)
SM (2) SMT202000399T1 (https=)
WO (1) WO2016205671A1 (https=)

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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
LT3505174T (lt) * 2015-06-17 2020-09-10 The Trustees Of Columbia University In The City Of New York Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus
WO2017112701A1 (en) 2015-12-22 2017-06-29 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
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SG11201900975XA (en) 2016-08-24 2019-03-28 Zogenix International Ltd 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
CA3096264A1 (en) * 2018-04-12 2019-10-17 Modis Therapeutics Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
CN112312929A (zh) 2018-04-18 2021-02-02 纽约市哥伦比亚大学理事会 用于包括线粒体dna耗竭综合征在内的由不平衡的核苷酸库引起的疾病的基因疗法
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
CN114929240B (zh) * 2019-08-19 2024-10-29 周格尼克斯公司 脱氧胞苷的多晶型形式、包含其的组合物和用途
IL322998A (en) * 2019-09-05 2025-10-01 Mitorainbow Therapeutics Inc Treatment of mitochondrial DNA depletion disorders
JP2023518395A (ja) 2020-03-19 2023-05-01 インテリア セラピューティクス，インコーポレイテッド 指向性ゲノム編集のための方法及び組成物
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
US20240041910A1 (en) * 2020-12-28 2024-02-08 Yamasa Corporation Muscular atrophy inhibitor and method for inhibiting muscular atrophy
WO2022204555A1 (en) * 2021-03-26 2022-09-29 Zogenix Mds, 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
CA3261416A1 (en) * 2022-07-12 2024-01-18 Ucb Biosciences, Inc. METHODS TO INCREASE THE BIOAVAILABILITY OF NUCLEOSID 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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CN101120947B (zh) * 1998-08-10 2010-11-24 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
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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
US11337980B2 (en) * 2015-06-05 2022-05-24 Fundació Hospital Universitari Vall D'hebron-Institut De Recerca Treatment of mitochondrial diseases
LT3505174T (lt) * 2015-06-17 2020-09-10 The Trustees Of Columbia University In The City Of New York Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus

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RU2020112901A (ru) 2020-06-05
RS59724B1 (sr) 2020-01-31
WO2016205671A1 (en) 2016-12-22
CN107847512A (zh) 2018-03-27
ES2748556T3 (es) 2020-03-17
HUE046399T2 (hu) 2020-03-30
HRP20191794T1 (hr) 2020-02-07
SMT202000399T1 (it) 2020-09-10
KR20220018623A (ko) 2022-02-15
JP2018521993A (ja) 2018-08-09
HRP20200949T1 (hr) 2020-10-02
US11666592B2 (en) 2023-06-06
KR20180039624A (ko) 2018-04-18
US20250360154A1 (en) 2025-11-27
EP3505174B1 (en) 2020-04-29
KR20250048602A (ko) 2025-04-09
MX2017016425A (es) 2018-12-11
JP7036782B2 (ja) 2022-03-15
SI3310362T1 (sl) 2020-02-28
JP6599484B2 (ja) 2019-10-30
US12251392B2 (en) 2025-03-18
IL275255A (en) 2020-07-30
AU2016280293A1 (en) 2018-02-08
CA2989653A1 (en) 2016-12-22
EP3505174A1 (en) 2019-07-03
JP2020019791A (ja) 2020-02-06
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IL275256A (en) 2020-07-30
CY1122605T1 (el) 2021-03-12
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US20210244754A1 (en) 2021-08-12
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IL256331B (en) 2020-08-31
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BR122020021913B1 (pt) 2021-07-06
HUE050678T2 (hu) 2020-12-28
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