EA201500699A1 - Агенты для лечения генетических заболеваний, возникающих в результате нонсенс-мутаций, и способы идентификации этих агентов - Google Patents
Агенты для лечения генетических заболеваний, возникающих в результате нонсенс-мутаций, и способы идентификации этих агентовInfo
- Publication number
- EA201500699A1 EA201500699A1 EA201500699A EA201500699A EA201500699A1 EA 201500699 A1 EA201500699 A1 EA 201500699A1 EA 201500699 A EA201500699 A EA 201500699A EA 201500699 A EA201500699 A EA 201500699A EA 201500699 A1 EA201500699 A1 EA 201500699A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- agents
- treatment
- methods
- mutations
- nonsens
- Prior art date
Links
Classifications
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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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
- A61K31/7048—Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin, digitoxin or digoxin
-
- 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
- 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
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
- A61P27/12—Ophthalmic agents for cataracts
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- 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
-
- 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
- G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
- G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
- G01N33/502—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing non-proliferative effects
- G01N33/5023—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing non-proliferative effects on expression patterns
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2500/00—Screening for compounds of potential therapeutic value
- G01N2500/10—Screening for compounds of potential therapeutic value involving cells
-
- 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
- G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
- G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
Abstract
Лечение генетических нейродегенеративных или нейроонтогенетических заболеваний, которые вызваны или связаны с нонсенс-мутациями или кодонами преждевременной терминации, с применением макролидов. Дополнительно описывают способы идентификации агентов, которые индуцируют сквозное пропитывание нонсенс-мутаций и кодонов с преждевременной терминацией, и их применение.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261745651P | 2012-12-24 | 2012-12-24 | |
US201361762900P | 2013-02-10 | 2013-02-10 | |
PCT/IL2013/051058 WO2014102778A2 (en) | 2012-12-24 | 2013-12-24 | Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same |
Publications (1)
Publication Number | Publication Date |
---|---|
EA201500699A1 true EA201500699A1 (ru) | 2015-12-30 |
Family
ID=49956287
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA201500699A EA201500699A1 (ru) | 2012-12-24 | 2013-12-24 | Агенты для лечения генетических заболеваний, возникающих в результате нонсенс-мутаций, и способы идентификации этих агентов |
Country Status (16)
Country | Link |
---|---|
US (1) | US10987370B2 (ru) |
EP (1) | EP2934548A2 (ru) |
JP (1) | JP2016503051A (ru) |
KR (1) | KR20150098675A (ru) |
AU (1) | AU2013368805A1 (ru) |
BR (1) | BR112015015075A2 (ru) |
CA (1) | CA2894992A1 (ru) |
CL (1) | CL2015001814A1 (ru) |
CR (1) | CR20150381A (ru) |
EA (1) | EA201500699A1 (ru) |
IL (1) | IL239467A0 (ru) |
MD (1) | MD20150056A2 (ru) |
MX (1) | MX2015008146A (ru) |
PE (1) | PE20151296A1 (ru) |
SG (1) | SG11201504933WA (ru) |
WO (1) | WO2014102778A2 (ru) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US10987370B2 (en) | 2012-12-24 | 2021-04-27 | Ramot At Tel-Aviv University Ltd. | Methods of inducing read-through of a nonsense mutation associated with ataxia telangiectasia, Rett syndrome or spinal muscular atrophy by erythromycin or azithromycin |
WO2015192198A1 (en) * | 2014-06-19 | 2015-12-23 | The Hospital For Sick Children | Treatment of peroxisome biogenesis disorder |
WO2015198329A1 (en) | 2014-06-25 | 2015-12-30 | Bio Blast Pharma Ltd. | Injectable formulations for intrathecal administration of antibiotic agents |
JP6460552B2 (ja) * | 2015-07-14 | 2019-01-30 | 国立大学法人大阪大学 | 筋強直性ジストロフィー治療薬 |
CA3005434A1 (en) | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
US11213537B2 (en) | 2017-09-18 | 2022-01-04 | Friedrich Miescher Institute For Biomedical Research | Inhibition of autism spectrum disorder using ribosomal read-through compounds |
WO2023235380A1 (en) * | 2022-06-01 | 2023-12-07 | Zikani Therapeutics, Inc. | Macrolides for treating genetic diseases |
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IT1176332B (it) | 1984-06-27 | 1987-08-18 | Erba Farmitalia | Procedimento per al preparazione di peptidi |
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CA2490062A1 (en) * | 2002-05-23 | 2003-12-04 | Activbiotics, Inc. | Methods of treating bacterial infections and diseases associated therewith |
WO2004001010A2 (en) | 2002-06-21 | 2003-12-31 | Ptc Therapeutics, Inc. | METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY |
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EP1765810B1 (en) * | 2004-06-08 | 2016-08-17 | Families of Spinal Muscular Atrophy | 2,4-diaminoquinazolines for the treatment of spinal muscular atrophy |
US20060018921A1 (en) * | 2004-07-16 | 2006-01-26 | Baylor College Of Medicine | Histone deacetylase inhibitors and cognitive applications |
JP2008527002A (ja) * | 2005-01-13 | 2008-07-24 | サートリス ファーマシューティカルズ, インコーポレイテッド | 神経変性障害および血液凝固障害を予防および処置するための新規組成物 |
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MX352861B (es) * | 2011-12-30 | 2017-12-13 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
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MX354074B (es) * | 2012-02-10 | 2018-02-12 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
BR112014021531B1 (pt) * | 2012-03-01 | 2022-10-04 | Ptc Therapeutics, Inc. | Composto, composição farmacêutica e usos dos mesmos |
US9914722B2 (en) * | 2012-03-23 | 2018-03-13 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
WO2014044972A1 (fr) * | 2012-09-19 | 2014-03-27 | Centre National De La Recherche Scientifique - Cnrs - | Traitement des neuronopathies motrices |
US10987370B2 (en) | 2012-12-24 | 2021-04-27 | Ramot At Tel-Aviv University Ltd. | Methods of inducing read-through of a nonsense mutation associated with ataxia telangiectasia, Rett syndrome or spinal muscular atrophy by erythromycin or azithromycin |
-
2013
- 2013-12-24 US US14/652,368 patent/US10987370B2/en active Active
- 2013-12-24 BR BR112015015075A patent/BR112015015075A2/pt not_active IP Right Cessation
- 2013-12-24 CA CA2894992A patent/CA2894992A1/en not_active Abandoned
- 2013-12-24 WO PCT/IL2013/051058 patent/WO2014102778A2/en active Application Filing
- 2013-12-24 EA EA201500699A patent/EA201500699A1/ru unknown
- 2013-12-24 JP JP2015548887A patent/JP2016503051A/ja active Pending
- 2013-12-24 KR KR1020157020037A patent/KR20150098675A/ko not_active Application Discontinuation
- 2013-12-24 MD MDA20150056A patent/MD20150056A2/ru not_active Application Discontinuation
- 2013-12-24 MX MX2015008146A patent/MX2015008146A/es unknown
- 2013-12-24 AU AU2013368805A patent/AU2013368805A1/en not_active Abandoned
- 2013-12-24 SG SG11201504933WA patent/SG11201504933WA/en unknown
- 2013-12-24 EP EP13821190.9A patent/EP2934548A2/en not_active Withdrawn
- 2013-12-24 PE PE2015001190A patent/PE20151296A1/es not_active Application Discontinuation
-
2015
- 2015-06-17 IL IL239467A patent/IL239467A0/en unknown
- 2015-06-23 CL CL2015001814A patent/CL2015001814A1/es unknown
- 2015-07-20 CR CR20150381A patent/CR20150381A/es unknown
Also Published As
Publication number | Publication date |
---|---|
US20150328247A1 (en) | 2015-11-19 |
AU2013368805A1 (en) | 2015-08-13 |
IL239467A0 (en) | 2015-07-30 |
CR20150381A (es) | 2015-09-09 |
KR20150098675A (ko) | 2015-08-28 |
CL2015001814A1 (es) | 2015-10-23 |
BR112015015075A2 (pt) | 2019-01-15 |
WO2014102778A3 (en) | 2014-08-21 |
MD20150056A2 (ru) | 2015-09-30 |
EP2934548A2 (en) | 2015-10-28 |
CA2894992A1 (en) | 2014-07-03 |
MX2015008146A (es) | 2016-01-20 |
JP2016503051A (ja) | 2016-02-01 |
PE20151296A1 (es) | 2015-10-15 |
WO2014102778A2 (en) | 2014-07-03 |
US10987370B2 (en) | 2021-04-27 |
SG11201504933WA (en) | 2015-07-30 |
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