SI3310362T1 - Deoksinukleozidna terapija za bolezni, povzročene z neuravnoteženimi nabori nukleotidov, vključno s sindromom pomanjkanja mitohondrijske DNA - Google Patents

Deoksinukleozidna terapija za bolezni, povzročene z neuravnoteženimi nabori nukleotidov, vključno s sindromom pomanjkanja mitohondrijske DNA

Info

Publication number
SI3310362T1
SI3310362T1 SI201630439T SI201630439T SI3310362T1 SI 3310362 T1 SI3310362 T1 SI 3310362T1 SI 201630439 T SI201630439 T SI 201630439T SI 201630439 T SI201630439 T SI 201630439T SI 3310362 T1 SI3310362 T1 SI 3310362T1
Authority
SI
Slovenia
Prior art keywords
diseases caused
mitochondrial dna
dna depletion
including mitochondrial
nucleotide pools
Prior art date
Application number
SI201630439T
Other languages
English (en)
Inventor
Michio Hirano
Caterina Garone
Ramon Marti
Original Assignee
The Trustees Of Columbia University In The City Of New York,
Fundacio 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,, Fundacio Hospital Universitari Vall D'hebron - Institut De Recerca filed Critical The Trustees Of Columbia University In The City Of New York,
Publication of SI3310362T1 publication Critical patent/SI3310362T1/sl

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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
    • A61K31/33—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
    • 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
    • A61K31/70—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
    • 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
    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6827—Hybridisation assays for detection of mutation or polymorphism
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
SI201630439T 2015-06-17 2016-06-17 Deoksinukleozidna terapija za bolezni, povzročene z neuravnoteženimi nabori nukleotidov, vključno s sindromom pomanjkanja mitohondrijske DNA SI3310362T1 (sl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17
EP16812537.5A EP3310362B1 (en) 2015-06-17 2016-06-17 Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes
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)

Publication Number Publication Date
SI3310362T1 true SI3310362T1 (sl) 2020-02-28

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
SI201630830T SI3505174T1 (sl) 2015-06-17 2016-06-17 Deoksinukleozidna terapija za bolezni, ki jih povzročajo neuravnotežene nukleotidne zbirke, vključno s sindromi deplecije mitohondrijske dnk
SI201630439T SI3310362T1 (sl) 2015-06-17 2016-06-17 Deoksinukleozidna terapija za bolezni, povzročene z neuravnoteženimi nabori nukleotidov, vključno s sindromom pomanjkanja mitohondrijske DNA

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SI201630830T SI3505174T1 (sl) 2015-06-17 2016-06-17 Deoksinukleozidna terapija za bolezni, ki jih povzročajo neuravnotežene nukleotidne zbirke, vključno s sindromi deplecije mitohondrijske dnk

Country Status (23)

Country Link
US (4) US10471087B2 (sl)
EP (3) EP3569236A1 (sl)
JP (3) JP6599484B2 (sl)
KR (2) KR20220018623A (sl)
CN (2) CN107847512A (sl)
AU (3) AU2016280293B2 (sl)
BR (2) BR122020021913B1 (sl)
CA (1) CA2989653A1 (sl)
CY (2) CY1122605T1 (sl)
DK (2) DK3310362T3 (sl)
ES (2) ES2808148T3 (sl)
HK (1) HK1252133A1 (sl)
HR (2) HRP20191794T1 (sl)
HU (2) HUE050678T2 (sl)
IL (3) IL275256B2 (sl)
LT (2) LT3310362T (sl)
MX (2) MX2020000269A (sl)
PL (2) PL3505174T3 (sl)
PT (2) PT3505174T (sl)
RS (2) RS59724B1 (sl)
RU (2) RU2721492C2 (sl)
SI (2) SI3505174T1 (sl)
WO (1) WO2016205671A1 (sl)

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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
RU2721492C2 (ru) * 2015-06-17 2020-05-19 Зе Трастис Оф Коламбия Юниверсити Ин Зе Сити Оф Нью-Йорк Дезоксинуклеозидная терапия заболеваний, вызванных несбалансированными пулами нуклеотидов, в том числе синдромов истощения митохондриальной днк
EP3393470B1 (en) 2015-12-22 2021-01-20 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
CN108883399B (zh) 2015-12-22 2021-06-22 周格尼克斯国际有限公司 芬氟拉明组合物及其制备方法
JP2019526544A (ja) 2016-08-24 2019-09-19 ゾゲニクス インターナショナル リミテッド ５−ｈｔ２ｂアゴニストの形成を阻害するための製剤およびその使用方法
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
JP2021526507A (ja) 2018-05-11 2021-10-07 ゾゲニクス インターナショナル リミテッド 発作により誘発される突然死を処置するための組成物および方法
EP4017502A4 (en) * 2019-08-19 2023-11-01 Modis Therapeutics Inc. POLYMORPH FORMS OF DEOXYCYTIDINE, COMPOSITIONS COMPRISING THEM AND USES
JP2022546611A (ja) * 2019-09-05 2022-11-04 ミトレインボー セラピューティクス，インコーポレーテッド ミトコンドリアｄｎａ枯渇障害の処置
KR20230003467A (ko) 2020-03-19 2023-01-06 인텔리아 테라퓨틱스, 인크. 지시된 게놈 편집을 위한 방법 및 조성물
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
EP4268829A1 (en) * 2020-12-28 2023-11-01 Yamasa Corporation Muscular atrophy inhibitor and method for inhibiting muscular atrophy
CA3209159A1 (en) * 2021-03-26 2022-09-29 Paul Glidden Aqueous solutions containing purines and pyrimidines and uses thereof
IL309421A (en) * 2021-06-18 2024-02-01 Zogenix Inc Deoxynucleoside preparations for the 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

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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
DE60037578T2 (de) * 1999-02-23 2009-01-08 The Regents Of The University Of California, Oakland Verwendung von triacetyluridin zur behandlung von mitochondrialen erkrankungen
ATE275406T1 (de) * 2000-05-26 2004-09-15 Idenix Cayman Ltd Methoden zur behandlung von delta hepatitis virus infektionen mit beta-l-2' deoxy-nucleosiden
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
MX2017015566A (es) * 2015-06-05 2018-08-15 Fundacio Hospital Univ Vall Dhebron Institut De Recerca Tratamiento de enfermedades mitocondriales.
RU2721492C2 (ru) * 2015-06-17 2020-05-19 Зе Трастис Оф Коламбия Юниверсити Ин Зе Сити Оф Нью-Йорк Дезоксинуклеозидная терапия заболеваний, вызванных несбалансированными пулами нуклеотидов, в том числе синдромов истощения митохондриальной днк

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JP2018521993A (ja) 2018-08-09
EP3569236A1 (en) 2019-11-20
RU2721492C2 (ru) 2020-05-19
CY1123107T1 (el) 2021-10-29
EP3505174B1 (en) 2020-04-29
IL275255B (en) 2021-04-29
WO2016205671A1 (en) 2016-12-22
AU2021250841B2 (en) 2023-02-09
BR112017027079B1 (pt) 2021-04-27
US20180133241A1 (en) 2018-05-17
AU2016280293B2 (en) 2020-07-02
SI3505174T1 (sl) 2021-02-26
EP3505174A1 (en) 2019-07-03
RU2018101305A (ru) 2019-07-17
MX2017016425A (es) 2018-12-11
CA2989653A1 (en) 2016-12-22
AU2021250841A1 (en) 2021-11-04
KR20180039624A (ko) 2018-04-18
IL256331A (en) 2018-02-28
EP3310362B1 (en) 2019-08-28
PL3310362T3 (pl) 2020-04-30
JP6599484B2 (ja) 2019-10-30
RU2018101305A3 (sl) 2019-10-31
JP6675037B2 (ja) 2020-04-01
AU2020204042A1 (en) 2020-07-09
ES2808148T3 (es) 2021-02-25
US10471087B2 (en) 2019-11-12
RU2020112901A (ru) 2020-06-05
DK3505174T3 (da) 2020-07-13
PT3310362T (pt) 2019-10-29
IL256331B (en) 2020-08-31
IL275255A (en) 2020-07-30
MX2020000269A (es) 2022-01-03
JP2020019792A (ja) 2020-02-06
IL275256B1 (en) 2023-05-01
CN116726035A (zh) 2023-09-12
WO2016205671A9 (en) 2018-01-04
EP3310362A1 (en) 2018-04-25
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
ES2748556T3 (es) 2020-03-17
CY1122605T1 (el) 2021-03-12
BR122020021913B1 (pt) 2021-07-06
CN107847512A (zh) 2018-03-27
HUE050678T2 (hu) 2020-12-28
HRP20200949T1 (hr) 2020-10-02
EP3310362A4 (en) 2018-12-19
JP2020019791A (ja) 2020-02-06
US20230277577A1 (en) 2023-09-07
US20200038424A1 (en) 2020-02-06
US11110111B2 (en) 2021-09-07
RS59724B1 (sr) 2020-01-31
DK3310362T3 (da) 2019-10-07
US11666592B2 (en) 2023-06-06
JP7036782B2 (ja) 2022-03-15
AU2016280293A1 (en) 2018-02-08
LT3310362T (lt) 2019-11-11
IL275256A (en) 2020-07-30
AU2020204042B2 (en) 2021-07-15
IL275256B2 (en) 2023-09-01
RS60572B1 (sr) 2020-08-31
KR20220018623A (ko) 2022-02-15
HRP20191794T1 (hr) 2020-02-07
HUE046399T2 (hu) 2020-03-30
PL3505174T3 (pl) 2020-08-24
LT3505174T (lt) 2020-09-10
HK1252133A1 (zh) 2019-05-17

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