CY1126058T1 - Αγωγη μιτοχονδριακων παθησεων - Google Patents
Αγωγη μιτοχονδριακων παθησεωνInfo
- Publication number
- CY1126058T1 CY1126058T1 CY20231100251T CY231100251T CY1126058T1 CY 1126058 T1 CY1126058 T1 CY 1126058T1 CY 20231100251 T CY20231100251 T CY 20231100251T CY 231100251 T CY231100251 T CY 231100251T CY 1126058 T1 CY1126058 T1 CY 1126058T1
- Authority
- CY
- Cyprus
- Prior art keywords
- treatment
- mitochondrial diseases
- mitochondrial dna
- caused
- syndrome
- Prior art date
Links
- 208000012268 mitochondrial disease Diseases 0.000 title 1
- 239000005549 deoxyribonucleoside Substances 0.000 abstract 2
- 208000011580 syndromic disease Diseases 0.000 abstract 2
- 108020005196 Mitochondrial DNA Proteins 0.000 abstract 1
- 206010059396 Mitochondrial DNA depletion Diseases 0.000 abstract 1
- 230000007547 defect Effects 0.000 abstract 1
- 230000037430 deletion Effects 0.000 abstract 1
- 238000012217 deletion Methods 0.000 abstract 1
- 230000004060 metabolic process Effects 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 235000011178 triphosphate Nutrition 0.000 abstract 1
- 239000001226 triphosphate Substances 0.000 abstract 1
- UNXRWKVEANCORM-UHFFFAOYSA-N triphosphoric acid Chemical compound OP(O)(=O)OP(O)(=O)OP(O)(O)=O UNXRWKVEANCORM-UHFFFAOYSA-N 0.000 abstract 1
Classifications
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C1/00—Preparation of hydrocarbons from one or more compounds, none of them being a hydrocarbon
-
- 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
- A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
- A61K31/52—Purines, e.g. adenine
-
- 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
- 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
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
Abstract
Η παρούσα εφεύρεση παρέχει σύνθεση περιλαμβάνουσα έναν ή περισσότερους δεοξυριβονουκλεοζίτες για χρήση στην αγωγή συνδρόμου εξάντλησης και/ή πολλαπλών διαγραφών μιτοχονδριακού DNA με την προϋπόθεση ότι το σύνδρομο δεν προκαλείται από ατέλεια στον μεταβολισμό τριφωσφορικών δεοξυριβονουκλεοζιτών (dNTP). Με τη χρήση της εφεύρεσης προκαλείται αποκατάσταση των επιπέδων μιτοχονδριακού DNA ανεξαρτήτως της σοβαρότητας της πάθησης του ασθενή, κάτι που συνεπάγεται η εφεύρεση να έχει υψηλή θεραπευτική αξία.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP15170825 | 2015-06-05 | ||
PCT/EP2016/062636 WO2016193421A1 (en) | 2015-06-05 | 2016-06-03 | Treatment of mitochondrial diseases |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1126058T1 true CY1126058T1 (el) | 2023-11-15 |
Family
ID=53298228
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20231100251T CY1126058T1 (el) | 2015-06-05 | 2023-05-26 | Αγωγη μιτοχονδριακων παθησεων |
Country Status (22)
Country | Link |
---|---|
US (2) | US11337980B2 (el) |
EP (2) | EP4218938A1 (el) |
JP (3) | JP6885544B2 (el) |
CN (1) | CN107820430A (el) |
AU (2) | AU2016273340B2 (el) |
BR (1) | BR112017025839A2 (el) |
CA (1) | CA2991271C (el) |
CY (1) | CY1126058T1 (el) |
DK (1) | DK3302499T3 (el) |
ES (1) | ES2944551T3 (el) |
FI (1) | FI3302499T3 (el) |
HR (1) | HRP20230277T1 (el) |
HU (1) | HUE062134T2 (el) |
IL (2) | IL255988B (el) |
LT (1) | LT3302499T (el) |
MX (2) | MX2017015566A (el) |
PL (1) | PL3302499T3 (el) |
PT (1) | PT3302499T (el) |
RS (1) | RS64273B1 (el) |
RU (1) | RU2745611C2 (el) |
SI (1) | SI3302499T1 (el) |
WO (1) | WO2016193421A1 (el) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN107820430A (zh) * | 2015-06-05 | 2018-03-20 | 瓦尔德西布伦大学医院基金会研究所 | 线粒体疾病的治疗 |
CN107847512A (zh) | 2015-06-17 | 2018-03-27 | 纽约市哥伦比亚大学理事会 | 用于包括线粒体dna耗竭综合症在内的由不平衡的核苷酸库引起的疾病的脱氧核苷疗法 |
Family Cites Families (10)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5246708A (en) | 1987-10-28 | 1993-09-21 | Pro-Neuron, Inc. | Methods for promoting wound healing with deoxyribonucleosides |
ZA892928B (en) * | 1988-04-25 | 1991-01-30 | Pro Neuron Inc | Pharmaceutical compositions containing deoxyribonucleosides for wound healing |
IT1247510B (it) * | 1991-04-19 | 1994-12-17 | Univ Cagliari | Composizioni terapeutiche ad attivita' anti-hiv costituite da 2',3'-didesossiadenosina e sostanze inibitrici della adenosina-deaminasi |
US20020006913A1 (en) * | 1997-11-04 | 2002-01-17 | Von Borstel Reid W. | Antimutagenic compositions for treatment and prevention of photodamage to skin |
US6472378B2 (en) * | 1998-08-31 | 2002-10-29 | Pro-Neuron, Inc. | Compositions and methods for treatment of mitochondrial diseases |
CN100379426C (zh) * | 1999-02-23 | 2008-04-09 | 加利福尼亚大学董事会 | 嘧啶核苷和嘧啶核苷酸前体的制药用途 |
WO2010126911A1 (en) * | 2009-04-28 | 2010-11-04 | Edison Pharmaceuticals, Inc. | Treatment of leber's hereditary optic neuropathy and dominant optic atrophy with tocotrienol quinones |
US20150065556A1 (en) * | 2013-08-05 | 2015-03-05 | Whitehead Institute For Biomedical Research | Therapeutic targets for mitochondrial disorders |
CN104524546B (zh) | 2014-12-18 | 2019-06-21 | 中国科学院广州生物医药与健康研究院 | 用于治疗Alpers-Huttenlocher综合征的药物组合物 |
CN107820430A (zh) * | 2015-06-05 | 2018-03-20 | 瓦尔德西布伦大学医院基金会研究所 | 线粒体疾病的治疗 |
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2016
- 2016-06-03 CN CN201680039230.6A patent/CN107820430A/zh active Pending
- 2016-06-03 JP JP2017562304A patent/JP6885544B2/ja active Active
- 2016-06-03 PT PT167288927T patent/PT3302499T/pt unknown
- 2016-06-03 RU RU2018123532A patent/RU2745611C2/ru active
- 2016-06-03 PL PL16728892.7T patent/PL3302499T3/pl unknown
- 2016-06-03 US US15/578,621 patent/US11337980B2/en active Active
- 2016-06-03 SI SI201631688T patent/SI3302499T1/sl unknown
- 2016-06-03 FI FIEP16728892.7T patent/FI3302499T3/fi active
- 2016-06-03 HU HUE16728892A patent/HUE062134T2/hu unknown
- 2016-06-03 RS RS20230259A patent/RS64273B1/sr unknown
- 2016-06-03 ES ES16728892T patent/ES2944551T3/es active Active
- 2016-06-03 BR BR112017025839A patent/BR112017025839A2/pt not_active Application Discontinuation
- 2016-06-03 EP EP23156608.4A patent/EP4218938A1/en active Pending
- 2016-06-03 EP EP16728892.7A patent/EP3302499B1/en active Active
- 2016-06-03 WO PCT/EP2016/062636 patent/WO2016193421A1/en active Application Filing
- 2016-06-03 AU AU2016273340A patent/AU2016273340B2/en active Active
- 2016-06-03 CA CA2991271A patent/CA2991271C/en active Active
- 2016-06-03 MX MX2017015566A patent/MX2017015566A/es unknown
- 2016-06-03 DK DK16728892.7T patent/DK3302499T3/da active
- 2016-06-03 HR HRP20230277TT patent/HRP20230277T1/hr unknown
- 2016-06-03 LT LTEPPCT/EP2016/062636T patent/LT3302499T/lt unknown
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2017
- 2017-11-29 IL IL255988A patent/IL255988B/en unknown
- 2017-12-01 MX MX2022012719A patent/MX2022012719A/es unknown
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2021
- 2021-04-28 JP JP2021075504A patent/JP2021121594A/ja active Pending
- 2021-12-08 AU AU2021282462A patent/AU2021282462B2/en active Active
-
2022
- 2022-01-10 IL IL289747A patent/IL289747A/en unknown
- 2022-04-25 US US17/728,698 patent/US20230060544A1/en active Pending
-
2023
- 2023-05-26 CY CY20231100251T patent/CY1126058T1/el unknown
- 2023-05-30 JP JP2023088479A patent/JP2023123444A/ja active Pending
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