CY1126058T1 - Αγωγη μιτοχονδριακων παθησεων - Google Patents

Αγωγη μιτοχονδριακων παθησεων

Info

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
Application number
CY20231100251T
Other languages
English (en)
Inventor
Ramon MARTÍ SEVES
Emiliano GONZÁLEZ VIOQUE
Cora BLÁZQUEZ BERMEJO
Javier TORRES TORRONTERAS
Raquel CABRERA PÉREZ
Yolanda CÁMARA NAVARRO
Original Assignee
Fundació 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 Fundació Hospital Universitari Vall D'hebron - Institut De Recerca filed Critical Fundació Hospital Universitari Vall D'hebron - Institut De Recerca
Publication of CY1126058T1 publication Critical patent/CY1126058T1/el

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C1/00Preparation of hydrocarbons from one or more compounds, none of them being a hydrocarbon
    • 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
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52Purines, e.g. adenine
    • 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
    • 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
    • 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
    • A61K31/70Carbohydrates; 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • 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

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.
CY20231100251T 2015-06-05 2023-05-26 Αγωγη μιτοχονδριακων παθησεων CY1126058T1 (el)

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

Also Published As

Publication number Publication date
IL255988B (en) 2022-02-01
ES2944551T3 (es) 2023-06-22
DK3302499T3 (da) 2023-04-03
AU2016273340B2 (en) 2021-09-09
CN107820430A (zh) 2018-03-20
EP3302499A1 (en) 2018-04-11
RU2745611C2 (ru) 2021-03-29
EP4218938A1 (en) 2023-08-02
JP2018516923A (ja) 2018-06-28
AU2021282462A1 (en) 2022-01-06
PT3302499T (pt) 2023-05-12
SI3302499T1 (sl) 2023-08-31
RU2018123532A (ru) 2019-12-30
US11337980B2 (en) 2022-05-24
LT3302499T (lt) 2023-05-25
RU2018123532A3 (el) 2019-12-30
IL289747A (en) 2022-03-01
HRP20230277T1 (hr) 2023-06-23
AU2016273340A1 (en) 2018-01-25
JP2023123444A (ja) 2023-09-05
RS64273B1 (sr) 2023-07-31
IL255988A (en) 2018-04-30
PL3302499T3 (pl) 2023-07-24
MX2022012719A (es) 2022-11-07
CA2991271A1 (en) 2016-12-08
AU2021282462B2 (en) 2023-10-05
MX2017015566A (es) 2018-08-15
HUE062134T2 (hu) 2023-09-28
JP2021121594A (ja) 2021-08-26
CA2991271C (en) 2023-10-17
JP6885544B2 (ja) 2021-06-16
WO2016193421A1 (en) 2016-12-08
EP3302499B1 (en) 2023-03-15
US20230060544A1 (en) 2023-03-02
FI3302499T3 (fi) 2023-05-04
US20180161332A1 (en) 2018-06-14
BR112017025839A2 (pt) 2018-08-14

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