LT3505174T - Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus - Google Patents

Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus

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Publication number
LT3505174T
LT3505174T LTEP19156021.8T LT19156021T LT3505174T LT 3505174 T LT3505174 T LT 3505174T LT 19156021 T LT19156021 T LT 19156021T LT 3505174 T LT3505174 T LT 3505174T
Authority
LT
Lithuania
Prior art keywords
mitochondress
syndromes
nucleotides
treatment
diseases caused
Prior art date
Application number
LTEP19156021.8T
Other languages
English (en)
Inventor
Michio Hirano
Caterina Garone
Ramon MARTI
Original Assignee
The Trustees Of Columbia University In The City Of New York
Fundaci Hospital Universitari Vall d`Hebron - Institut de Recerca
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 The Trustees Of Columbia University In The City Of New York, Fundaci Hospital Universitari Vall d`Hebron - Institut de Recerca filed Critical The Trustees Of Columbia University In The City Of New York
Publication of LT3505174T publication Critical patent/LT3505174T/lt

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7068Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/513Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7068Compounds 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
    • A61K31/7072Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7076Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7076Compounds 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
    • A61K31/708Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K9/0012Galenical forms characterised by the site of application
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813Hybridisation assays
    • C12Q1/6827Hybridisation assays for detection of mutation or polymorphism
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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  • Proteomics, Peptides & Aminoacids (AREA)
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LTEP19156021.8T 2015-06-17 2016-06-17 Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus LT3505174T (lt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17

Publications (1)

Publication Number Publication Date
LT3505174T true LT3505174T (lt) 2020-09-10

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
LTEP19156021.8T LT3505174T (lt) 2015-06-17 2016-06-17 Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus
LT16812537T LT3310362T (lt) 2015-06-17 2016-06-17 Deoksinukleozidų terapija, skirta ligoms, sukeltoms nesubalansuotų nukleotidų telkinių, įskaitant mitochondrijų dnr išeikvojimo sindromą

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LT16812537T LT3310362T (lt) 2015-06-17 2016-06-17 Deoksinukleozidų terapija, skirta ligoms, sukeltoms nesubalansuotų nukleotidų telkinių, įskaitant mitochondrijų dnr išeikvojimo sindromą

Country Status (24)

Country Link
US (5) US10471087B2 (lt)
EP (3) EP3505174B1 (lt)
JP (3) JP6599484B2 (lt)
KR (3) KR20250048602A (lt)
CN (2) CN107847512A (lt)
AU (3) AU2016280293B2 (lt)
BR (2) BR112017027079B1 (lt)
CA (1) CA2989653A1 (lt)
CY (2) CY1122605T1 (lt)
DK (2) DK3310362T3 (lt)
ES (2) ES2748556T3 (lt)
HK (1) HK1252133A1 (lt)
HR (2) HRP20191794T1 (lt)
HU (2) HUE046399T2 (lt)
IL (3) IL275256B2 (lt)
LT (2) LT3505174T (lt)
MX (2) MX2017016425A (lt)
PL (2) PL3505174T3 (lt)
PT (2) PT3505174T (lt)
RS (2) RS60572B1 (lt)
RU (1) RU2721492C2 (lt)
SI (2) SI3310362T1 (lt)
SM (2) SMT202000399T1 (lt)
WO (1) WO2016205671A1 (lt)

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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
ES3024232T3 (en) 2015-12-22 2025-06-04 Zogenix International Ltd Fenfluramine compositions and methods of preparing the same
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
EP1808177B1 (en) * 1999-02-23 2018-08-08 The Regents of The University of California Methods of treatment of mitochondrial disorders
EA005890B1 (ru) * 2000-05-26 2005-06-30 Айденикс (Кайман) Лимитед СПОСОБЫ ЛЕЧЕНИЯ ИНФЕКЦИЙ, ВЫЗЫВАЕМЫХ ВИРУСОМ ГЕПАТИТА ДЕЛЬТА, С ИСПОЛЬЗОВАНИЕМ β-L-2'-ДЕЗОКСИНУКЛЕОЗИДОВ
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
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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JP2020019792A (ja) 2020-02-06
BR112017027079B1 (pt) 2021-04-27
AU2020204042B2 (en) 2021-07-15
LT3310362T (lt) 2019-11-11
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
EP3310362B1 (en) 2019-08-28
IL275256A (en) 2020-07-30
CY1122605T1 (el) 2021-03-12
SMT201900571T1 (it) 2019-11-13
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
RU2018101305A (ru) 2019-07-17
IL256331B (en) 2020-08-31
CN116726035A (zh) 2023-09-12
WO2016205671A9 (en) 2018-01-04
IL275256B1 (en) 2023-05-01
BR122020021913B1 (pt) 2021-07-06
HUE050678T2 (hu) 2020-12-28
IL256331A (en) 2018-02-28
DK3310362T3 (da) 2019-10-07
EP3310362A1 (en) 2018-04-25
EP3310362A4 (en) 2018-12-19
MX389016B (es) 2025-03-20
US20180133241A1 (en) 2018-05-17
AU2016280293B2 (en) 2020-07-02
JP6675037B2 (ja) 2020-04-01
DK3505174T3 (da) 2020-07-13
EP3569236A1 (en) 2019-11-20
MX2020000269A (es) 2022-01-03
HK1252133A1 (zh) 2019-05-17
PL3310362T3 (pl) 2020-04-30
US10471087B2 (en) 2019-11-12
RU2721492C2 (ru) 2020-05-19
PL3505174T3 (pl) 2020-08-24
IL275256B2 (en) 2023-09-01
SI3505174T1 (sl) 2021-02-26
AU2021250841A1 (en) 2021-11-04
US20200038424A1 (en) 2020-02-06
IL275255B (en) 2021-04-29
US11110111B2 (en) 2021-09-07
CY1123107T1 (el) 2021-10-29
ES2808148T3 (es) 2021-02-25
AU2021250841B2 (en) 2023-02-09
AU2020204042A1 (en) 2020-07-09
RS60572B1 (sr) 2020-08-31
RU2018101305A3 (lt) 2019-10-31
PT3310362T (pt) 2019-10-29
US20230277577A1 (en) 2023-09-07

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