PE20151296A1 - Agentes para tratar enfermedades geneticas derivadas de mutaciones sin sentido y metodos para identificarlas - Google Patents
Agentes para tratar enfermedades geneticas derivadas de mutaciones sin sentido y metodos para identificarlasInfo
- Publication number
- PE20151296A1 PE20151296A1 PE2015001190A PE2015001190A PE20151296A1 PE 20151296 A1 PE20151296 A1 PE 20151296A1 PE 2015001190 A PE2015001190 A PE 2015001190A PE 2015001190 A PE2015001190 A PE 2015001190A PE 20151296 A1 PE20151296 A1 PE 20151296A1
- Authority
- PE
- Peru
- Prior art keywords
- neurodetic
- methods
- identify
- genetic diseases
- agents
- Prior art date
Links
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
- 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
SE REFIERE A METODOS PARA IDENTIFICAR AGENTES CAPACES DE INDUCIR LA TRANSLECTURA DE LAS MUTACIONES SIN SENTIDO QUE PROVOCAN LA FORMACION DE CODONES DE TERMINACION PREMATUROS, UTILES EN EL TRATAMIENTO DE LAS ENFERMEDADES GENETICAS NEURODEGENERATIVAS O DEL NEURODESARROLLO ASOCIADA CON UNA MUTACION SIN SENTIDO, TALES COMO ATROFIA MUSCULAR ESPINAL, ATAXIA-TELANGIECTASIA, SINDROME DE RETT, SINDROME DE USHER, ENTRE OTROS. EN UNA MODALIDAD, LOS AGENTES CAPACES DE INDUCIR LA TRANSLECTURA DE LAS MUTACIONES SIN SENTIDO SON MACROLIDOS ANTIBIOTICOS. TAMBIEN SE REFIERE A COMPOSICIONES QUE COMPRENDEN AL MENOS UN MACROLIDO ANTIBIOTICO, SELECCIONADO PREFERENTEMENTE ENTRE ERITROMICINA, AZITROMICINA Y CLARITROMICINA, O CUALQUIER COMBINACION DE AL MENOS DOS DE DICHOS MACROLIDOS. DICHAS COMPOSICIONES SE ADMINISTRAN PREFERENTEMENTE POR VIA INTRATECAL, INTRANEURAL, INTRACEREBRAL, INTRAVENTRICULAR E INTRACRANEAL
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261745651P | 2012-12-24 | 2012-12-24 | |
US201361762900P | 2013-02-10 | 2013-02-10 |
Publications (1)
Publication Number | Publication Date |
---|---|
PE20151296A1 true PE20151296A1 (es) | 2015-10-15 |
Family
ID=49956287
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2015001190A PE20151296A1 (es) | 2012-12-24 | 2013-12-24 | Agentes para tratar enfermedades geneticas derivadas de mutaciones sin sentido y metodos para identificarlas |
Country Status (16)
Country | Link |
---|---|
US (1) | US10987370B2 (es) |
EP (1) | EP2934548A2 (es) |
JP (1) | JP2016503051A (es) |
KR (1) | KR20150098675A (es) |
AU (1) | AU2013368805A1 (es) |
BR (1) | BR112015015075A2 (es) |
CA (1) | CA2894992A1 (es) |
CL (1) | CL2015001814A1 (es) |
CR (1) | CR20150381A (es) |
EA (1) | EA201500699A1 (es) |
IL (1) | IL239467A0 (es) |
MD (1) | MD20150056A2 (es) |
MX (1) | MX2015008146A (es) |
PE (1) | PE20151296A1 (es) |
SG (1) | SG11201504933WA (es) |
WO (1) | WO2014102778A2 (es) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2013368805A1 (en) | 2012-12-24 | 2015-08-13 | Ramot At Tel-Aviv University Ltd. | Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same |
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 |
WO2017010382A1 (ja) * | 2015-07-14 | 2017-01-19 | 国立大学法人大阪大学 | 筋強直性ジストロフィー治療薬 |
EP3377073A4 (en) | 2015-11-16 | 2019-06-26 | Ohio State Innovation Foundation | METHOD AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES WITH THE 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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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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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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CN104349676B (zh) * | 2011-10-31 | 2017-05-31 | 约翰霍普金斯大学 | 用于治疗自闭症的方法和组合物 |
BR112014016287B1 (pt) * | 2011-12-30 | 2022-08-09 | F. Hoffmann - La Roche Ag | Composto e composição farmacêutica |
CN104203239B (zh) * | 2012-01-26 | 2017-09-08 | Ptc医疗公司 | 用于治疗脊髓性肌萎缩的化合物 |
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KR102109992B1 (ko) * | 2012-03-23 | 2020-05-13 | 피티씨 테라퓨틱스, 인크. | 척수성 근위축증을 치료하기 위한 화합물 |
US20150238483A1 (en) * | 2012-09-19 | 2015-08-27 | Centre National De La Recherche Scientifique- Cnrs | Traitement des neuronopathies motrices |
AU2013368805A1 (en) | 2012-12-24 | 2015-08-13 | Ramot At Tel-Aviv University Ltd. | Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same |
-
2013
- 2013-12-24 AU AU2013368805A patent/AU2013368805A1/en not_active Abandoned
- 2013-12-24 MD MDA20150056A patent/MD20150056A2/ro not_active Application Discontinuation
- 2013-12-24 US US14/652,368 patent/US10987370B2/en active Active
- 2013-12-24 JP JP2015548887A patent/JP2016503051A/ja active Pending
- 2013-12-24 BR BR112015015075A patent/BR112015015075A2/pt not_active IP Right Cessation
- 2013-12-24 WO PCT/IL2013/051058 patent/WO2014102778A2/en active Application Filing
- 2013-12-24 EP EP13821190.9A patent/EP2934548A2/en not_active Withdrawn
- 2013-12-24 CA CA2894992A patent/CA2894992A1/en not_active Abandoned
- 2013-12-24 PE PE2015001190A patent/PE20151296A1/es not_active Application Discontinuation
- 2013-12-24 EA EA201500699A patent/EA201500699A1/ru unknown
- 2013-12-24 KR KR1020157020037A patent/KR20150098675A/ko not_active Application Discontinuation
- 2013-12-24 SG SG11201504933WA patent/SG11201504933WA/en unknown
- 2013-12-24 MX MX2015008146A patent/MX2015008146A/es unknown
-
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 |
---|---|
SG11201504933WA (en) | 2015-07-30 |
CR20150381A (es) | 2015-09-09 |
JP2016503051A (ja) | 2016-02-01 |
CA2894992A1 (en) | 2014-07-03 |
WO2014102778A2 (en) | 2014-07-03 |
BR112015015075A2 (pt) | 2019-01-15 |
MX2015008146A (es) | 2016-01-20 |
MD20150056A2 (ro) | 2015-09-30 |
WO2014102778A3 (en) | 2014-08-21 |
IL239467A0 (en) | 2015-07-30 |
AU2013368805A1 (en) | 2015-08-13 |
EP2934548A2 (en) | 2015-10-28 |
KR20150098675A (ko) | 2015-08-28 |
CL2015001814A1 (es) | 2015-10-23 |
US20150328247A1 (en) | 2015-11-19 |
EA201500699A1 (ru) | 2015-12-30 |
US10987370B2 (en) | 2021-04-27 |
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Legal Events
Date | Code | Title | Description |
---|---|---|---|
FD | Application declared void or lapsed |