CY1123107T1 - Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna - Google Patents

Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna

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Publication number
CY1123107T1
CY1123107T1 CY20201100578T CY201100578T CY1123107T1 CY 1123107 T1 CY1123107 T1 CY 1123107T1 CY 20201100578 T CY20201100578 T CY 20201100578T CY 201100578 T CY201100578 T CY 201100578T CY 1123107 T1 CY1123107 T1 CY 1123107T1
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CY
Cyprus
Prior art keywords
mitochondrial dna
dna depletion
deoxynucleoside
unbalanced nucleotide
diseases due
Prior art date
Application number
CY20201100578T
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English (en)
Inventor
Michio Hirano
Caterina Garone
Ramon MARTI
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The Trustees Of Columbia University In The City Of New York
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Application filed by The Trustees Of Columbia University In The City Of New York filed Critical The Trustees Of Columbia University In The City Of New York
Publication of CY1123107T1 publication Critical patent/CY1123107T1/el

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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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Abstract

Η εφεύρεση αφορά γενικά μία φαρμακολογική θεραπεία για ανθρώπινες γενετικές νόσους, συγκεκριμένα νόσους οι οποίες χαρακτηρίζονται από μη ισορροπημένες ομάδες νουκλεοτιδίων, πλέον συγκεκριμένα σύνδρομα εξάντλησης μιτοχονδριακού DNA και πλέον συγκεκριμένα, έλλειψη κινάσης θυμιδίνης 2 (ΤΚ2). Η φαρμακολογική θεραπεία συμπεριλαμβάνει τη χορήγηση τουλάχιστον ενός δεοξυνουκλεοσιδίου ή μειγμάτων αυτών. Για την αγωγή της έλλειψης ΤΚ2, η φαρμακολογική θεραπεία συμπεριλαμβάνει τη χορήγηση είτε δεοξυθυμιδίνης (dT) είτε δεοξυκυτιδίνης (dC) είτε μειγμάτων αυτών. Η χορήγηση αυτή δεοξυνουκλεοσιδίων μπορεί να εφαρμόζεται σε άλλες διαταραχές μη ισορροπημένων ομάδων νουκλεοτιδίων, ειδικά αυτών οι οποίες απαντώνται στο σύνδρομο εξάντλησης μιτοχονδριακού DNA.
CY20201100578T 2015-06-17 2020-06-25 Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna CY1123107T1 (el)

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

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CY1123107T1 true CY1123107T1 (el) 2021-10-29

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CY20191101127T CY1122605T1 (el) 2015-06-17 2019-10-30 Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna
CY20201100578T CY1123107T1 (el) 2015-06-17 2020-06-25 Θεραπεια δεοξυνουκλεοσιδιου για νοσους οι οποιες οφειλονται σε μη ισορροπημενες ομαδες νουκλεοτιδιων, οπου συμπεριλαμβανονται συνδρομα εξαντλησης μιτοχονδριακου dna

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US (4) US10471087B2 (el)
EP (3) EP3310362B1 (el)
JP (3) JP6599484B2 (el)
KR (2) KR20180039624A (el)
CN (2) CN116726035A (el)
AU (3) AU2016280293B2 (el)
BR (2) BR112017027079B1 (el)
CA (1) CA2989653A1 (el)
CY (2) CY1122605T1 (el)
DK (2) DK3505174T3 (el)
ES (2) ES2748556T3 (el)
HK (1) HK1252133A1 (el)
HR (2) HRP20191794T1 (el)
HU (2) HUE050678T2 (el)
IL (3) IL275256B2 (el)
LT (2) LT3505174T (el)
MX (2) MX2017016425A (el)
PL (2) PL3310362T3 (el)
PT (2) PT3310362T (el)
RS (2) RS60572B1 (el)
RU (2) RU2721492C2 (el)
SI (2) SI3310362T1 (el)
WO (1) WO2016205671A1 (el)

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WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
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EP3790537A1 (en) 2018-05-11 2021-03-17 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
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CN114340641A (zh) * 2019-09-05 2022-04-12 米托瑞因博治疗股份有限公司 治疗线粒体dna缺失障碍
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)
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WO2022145439A1 (ja) * 2020-12-28 2022-07-07 ヤマサ醤油株式会社 筋萎縮抑制剤及び筋萎縮を抑制させる方法
JP2024515025A (ja) * 2021-03-26 2024-04-04 ゾジェニックス,インコーポレイテッド プリン及びピリミジンを含有する水性溶液及びその使用
CN117500510A (zh) * 2021-06-18 2024-02-02 周格尼克斯公司 用于治疗由失衡的核苷酸池引起的线粒体疾病的脱氧核苷的前药
WO2024015453A1 (en) * 2022-07-12 2024-01-18 Zogenix Mds, Inc. Methods for increasing the bioavailability of nucleoside medicinal agents

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DK3505174T3 (da) * 2015-06-17 2020-07-13 Univ Columbia Deoxynukleosidterapi mod sygdomme forårsaget af ubalancerede nukleotid-pools, herunder mitokondrielt dna-depletionssyndromer

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LT3505174T (lt) 2020-09-10
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
JP2020019792A (ja) 2020-02-06
JP6599484B2 (ja) 2019-10-30
AU2021250841B2 (en) 2023-02-09
SI3310362T1 (sl) 2020-02-28
BR122020021913B1 (pt) 2021-07-06
RS59724B1 (sr) 2020-01-31
CN116726035A (zh) 2023-09-12
JP2020019791A (ja) 2020-02-06
US20230277577A1 (en) 2023-09-07
ES2748556T3 (es) 2020-03-17
HRP20200949T1 (hr) 2020-10-02
PT3310362T (pt) 2019-10-29
JP7036782B2 (ja) 2022-03-15
CY1122605T1 (el) 2021-03-12
CA2989653A1 (en) 2016-12-22
US11110111B2 (en) 2021-09-07
ES2808148T3 (es) 2021-02-25
RU2020112901A (ru) 2020-06-05
AU2021250841A1 (en) 2021-11-04
EP3505174B1 (en) 2020-04-29
EP3569236A1 (en) 2019-11-20
IL275255B (en) 2021-04-29
RU2018101305A (ru) 2019-07-17
KR20180039624A (ko) 2018-04-18
US20180133241A1 (en) 2018-05-17
KR20220018623A (ko) 2022-02-15
PL3505174T3 (pl) 2020-08-24
IL256331B (en) 2020-08-31
IL275256A (en) 2020-07-30
IL256331A (en) 2018-02-28
US11666592B2 (en) 2023-06-06
EP3310362B1 (en) 2019-08-28
HUE050678T2 (hu) 2020-12-28
EP3310362A1 (en) 2018-04-25
RU2721492C2 (ru) 2020-05-19
MX2020000269A (es) 2022-01-03
DK3505174T3 (da) 2020-07-13
RU2018101305A3 (el) 2019-10-31
SI3505174T1 (sl) 2021-02-26
AU2020204042B2 (en) 2021-07-15
JP6675037B2 (ja) 2020-04-01
HK1252133A1 (zh) 2019-05-17
IL275256B2 (en) 2023-09-01
US10471087B2 (en) 2019-11-12
AU2016280293B2 (en) 2020-07-02
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DK3310362T3 (da) 2019-10-07
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US20200038424A1 (en) 2020-02-06
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WO2016205671A1 (en) 2016-12-22
IL275255A (en) 2020-07-30
MX2017016425A (es) 2018-12-11
RS60572B1 (sr) 2020-08-31
BR112017027079B1 (pt) 2021-04-27
EP3310362A4 (en) 2018-12-19
LT3310362T (lt) 2019-11-11
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