PTC THERAPEUTICS, INC. Patent applications |
Patent application number | Title | Published |
20150344877 | METHODS FOR TREATING VIRAL INFECTIONS - The present invention relates to compounds that modulate ribosomal frameshifting and nucleic acid constructs for use in methods for identifying or validation such compounds. In particular, the present invention relates to the use of nucleic acid constructs to identify or validate compounds capable of modulating the efficiency of programmed ribosomal frameshifting and the use of said compounds to inhibit the replication or infectivity of viruses that employ programmed ribosomal frameshifting. | 12-03-2015 |
20150283115 | METHODS FOR TREATING SPINAL MUSCULAR ATROPHY - Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of said compounds to produce a stabilized SMNΔEx7 protein and treat Spinal Muscular Dystrophy. | 10-08-2015 |
20150274674 | PYRAZOLE OR TRIAZOLE COMPOUNDS AND THEIR USE FOR THE MANUFACTURE OF A MEDICAMENT FOR TREATING SOMATIC MUTATION-RELATED DISEASES - The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA. | 10-01-2015 |
20150253304 | Methods And Agents For Screening For Compounds Capable Of Modulating Gene Expression - The invention relates to the fields of screening assays, compounds, and methods for altering gene expression and protein levels. In particular, the invention includes assays to screen for agents capable of modulating gene expression in a UTR-dependent manner and agents capable of modulating gene expression. | 09-10-2015 |
20150148346 | COMPOSITIONS OF 1,2,4-OXADIAZOLE BENZOIC ACID COMPOUNDS AND METHODS FOR THEIR USE - Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith. | 05-28-2015 |
20150141418 | TETRAHYDROCARBAZOLES AS ACTIVE AGENTS FOR INHIBITING VEGF PRODUCTION BY TRANSLATIONAL CONTROL - The present invention relates to methods, compounds, and compositions for inhibiting angiogenesis. More particularly, the present invention relates to methods, compounds, and compositions for inhibiting VEGF production. | 05-21-2015 |
20150126515 | COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY - Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. | 05-07-2015 |
20150080362 | ANTIBACTERIAL COMPOUNDS AND METHODS FOR USE - The present description relates to compounds and forms and pharmaceutical compositions thereof and methods for use thereof to treat or ameliorate bacterial infections caused by wild-type and multi-drug resistant Gram-negative and Gram-positive pathogens. | 03-19-2015 |
20150038437 | ANTIBACTERIAL COMPOUNDS AND METHODS FOR USE - The present description relates to compounds and forms and pharmaceutical compositions thereof and methods for use thereof to treat or ameliorate bacterial infections caused by wild-type and multi-drug resistant Gram-negative and Gram-positive pathogens. | 02-05-2015 |
20140343009 | METHODS FOR TREATING METHYLMALONIC ACIDEMIA - Methods for treating methylmalonic acidemia in which at least one allele of a gene associated with MMA (e.g., the MUT, MMAA, or MMAB gene) contains a mutation (e.g., nonsense mutation) that results in a premature stop codon in RNA encoded by an allele of the gene associated with MMA involving the administration of a compound that promotes readthrough of RNA (e.g., messenger RNA) containing a premature stop codon encoded by an allele of the gene associated with MMA are described. The compound can be administered as a single-agent therapy or in combination with one or more additional therapies to a human in need of such treatment. | 11-20-2014 |
20140301978 | METHODS FOR USING 1,2,4-OXADIAZOLE BENZOIC ACID COMPOUNDS - Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith. | 10-09-2014 |
20140235679 | CRYSTALLINE FORMS OF 3-[5-(2-FLUOROPHENYL)-[1,2,4]OXADIAZOL-3-YL]-BENZOIC ACID - The present invention relates to crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid, pharmaceutical compositions and dosage forms comprising the crystalline forms, methods of making the crystalline forms and methods for their use for the treatment, prevention or management of diseases ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay. | 08-21-2014 |
20140235586 | COMPOUNDS FOR NONSENSE SUPPRESSION, AND METHODS FOR THEIR USE - The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA. | 08-21-2014 |
20140221436 | COMPOSITIONS FOR AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR THE TREATMENT OF DISEASE - The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations. | 08-07-2014 |
20140135305 | METHODS FOR TREATING SPINAL MUSCULAR ATROPHY - The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA. | 05-15-2014 |
20130347133 | Methods For Identifying Compounds That Modulate Untranslated Region-Dependent Gene Expression And Methods Of Using Same - The present invention relates to methods for identifying compounds that modulate untranslated region-dependent expression of a target gene. The invention particularly relates to using untranslated regions of a target gene or fragments thereof linked to a reporter gene to identify compounds that modulate untranslated region-dependent expression of a target gene. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads. | 12-26-2013 |
20130289007 | METHODS FOR USING 1,2,4-OXADIAZOLE BENZOIC ACID COMPOUNDS - Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith. | 10-31-2013 |
20130171103 | METHODS FOR TREATING VIRAL CONDITIONS - Compounds that selectively inhibit viral replication or the production of viral RNA or DNA, viral protein or virus induced cytopathic effects and compositions comprising such Compounds are described. Also described are methods of inhibiting viral replication or the production of viral RNA or DNA, viral protein or virus induced cytopathic effects using such Compounds and methods for treating viral infections involving the administration of such Compounds. The Compounds may be administered as a single agent therapy or in combination with one or more additional therapies to a human in need of such treatments. | 07-04-2013 |
20130158081 | CRYSTALLINE FORMS OF 3-[5-(2-FLUOROPHENYL)-[1,2,4]OXADIAZOL-3-YL]-BENZOIC ACID - The present invention relates to crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid, pharmaceutical compositions and dosage forms comprising the crystalline forms, methods of making the crystalline forms and methods for their use for the treatment, prevention or management of diseases ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay. | 06-20-2013 |
20120202763 | METHODS FOR TREATING CANCER AND NON-NEOPLASTIC CONDITIONS - Compounds that selectively inhibit pathological production of human vascular endothelial growth factor (VEGF) and compositions comprising such Compounds are described. Compounds that inhibit viral replication or the production of viral RNA or DNA or viral protein and compositions comprising such Compounds are described. Also described are methods of reducing VEGF using such Compounds and methods for treating cancer and non-neoplastic conditions involving the administration of such Compounds. Further described are methods of inhibiting viral replication or the production of viral RNA or DNA or viral protein using such Compounds and methods for treating viral infections involving the administration of such Compounds. The Compounds may be administered as a single agent therapy or in combination with one or more additional therapies to a human in need of such treatments. | 08-09-2012 |
20120184574 | INDOLE DERIVATIVES AND METHODS FOR ANTIVIRAL TREATMENT - The present invention is directed to compounds and forms and pharmaceutical compositions thereof useful for treating a viral infection, or for affecting viral activity by modulating viral replication. | 07-19-2012 |
20120135999 | COMPOUNDS AND METHODS FOR ANTIVIRAL TREATMENT - The present invention is directed to compounds and forms and pharmaceutical compositions thereof useful for treating a viral infection, or for affecting viral activity by modulating viral replication. | 05-31-2012 |
20120087893 | COMBINATIONS OF A HCV INHIBITOR SUCH AS BICYCLIC PYRROLE DERIVATIVES AND A THERAPEUTIC AGENT - The present invention is directed to a combination product for treating or ameliorating hepatitis C virus (HCV) infection or disorders or symptoms associated therewith in a subject in need thereof comprising, a HCV inhibitor and one or more therapeutic agents selected from either or both a HCV protease inhibitor and one or more different therapeutic agents administered in combination in an effective amount to the subject. | 04-12-2012 |
20120070412 | HCV Inhibitor and Therapeutic Agent Combinations - The present invention is directed to a combination product for treating or ameliorating hepatitis C virus (HCV) infection or disorders or symptoms associated therewith in a subject in need thereof comprising, a HCV inhibitor and one or more therapeutic agents selected from either or both a HCV protease inhibitor and one or more different therapeutic agents administered in combination in an effective amount to the subject. | 03-22-2012 |
20120027721 | HCV Inhibitor and Therapeutic Agent Combinations - The present invention is directed to a combination product for treating or ameliorating hepatitis C virus (HCV) infection or disorders or symptoms associated therewith in a subject in need thereof comprising, a HCV inhibitor and one or more therapeutic agents selected from either or both a HCV protease inhibitor and one or more different therapeutic agents administered in combination in an effective amount to the subject. | 02-02-2012 |
20120009142 | METHODS FOR TREATING HEPATITIS C - In accordance with the present invention, compounds that inhibit viral replication, preferably Hepatitis C Virus (HCV) replication, have been identified, and methods for their use provided. In one aspect of the invention, compounds useful in the treatment or prevention of a viral infection are provided. In another aspect of the invention, compounds useful in the treatment or prevention of HCV infection are provided. | 01-12-2012 |
20110268698 | METHODS FOR TREATING HEPATITIS C - The present invention provides compounds, pharmaceutical compositions, and methods of using such compounds or compositions for treating infection by a virus, or for affecting viral IBES activity. | 11-03-2011 |
20110190239 | BMI-1 PROTEIN EXPRESSION MODULATORS - The compounds, pharmaceutical compositions, and methods of using such compounds or compositions thereof described herein are useful for treating a disease modulated by B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein expression. | 08-04-2011 |
20110165581 | METHODS FOR SCREENING FOR COMPOUNDS FOR TREATING CANCER - The methods described herein provide nucleic acid constructs and screening methods for identifying and validating compounds for use in the treatment of cancer, wherein the compounds down-regulate the post-transcriptional expression of Bmi-1. | 07-07-2011 |
20110160164 | COMPOUNDS FOR NONSENSE SUPPRESSION AND METHODS FOR THEIR USE - The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA. | 06-30-2011 |
20100305100 | METHODS FOR TREATING HEPATITIS C - The present invention provides compounds, pharmaceutical compositions, and methods of using such compounds or compositions for treating infection by a virus, or for affecting viral IRES activity. | 12-02-2010 |
20100292187 | METHODS FOR TREATING HEPATITIS C - The present invention provides compounds, pharmaceutical compositions, and methods of using such compounds or compositions for treating infection by a virus, or for affecting viral IRES activity. | 11-18-2010 |
20100099672 | METHODS FOR TREATING HEPATITIS C - In accordance with the present invention, compounds that can inhibit viral replication, preferably Hepatitis C Virus (HCV) replication, have been identified, and methods for their use provided. In one aspect of the invention, compounds useful in the treatment or prevention of a viral infection are provided. In another aspect of the invention, compounds useful in the treatment or prevention of HCV infection are provided. | 04-22-2010 |
20090203752 | COMPOUNDS FOR NONSENSE SUPPRESSION, AND METHODS FOR THEIR USE - The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA. | 08-13-2009 |
20080318222 | Cell Based Methods And Systems For The Identification Of Rna Regulatory Sequences And Compounds That Modulate Their Functions - The present invention is directed to an in vivo method for screening for and/or identifying RNA regulatory sequences involved in translational control. The method includes: culturing cells including: (i) a translational system; (ii) a reporter mRNA; and (iii) an RNA test sequence under suitable conditions for translation of the reporter mRNA, wherein the RNA test sequence is not attached to the reporter mRNA. The method also includes measuring the effect of the test sequence on the translation of the reporter mRNA, wherein a test sequence that modifies the translation of the reporter mRNA includes an RNA regulatory element. The invention also provides methods and systems for screening for and/or identifying test compounds that modulate the regulatory activity of an RNA regulatory sequence. This method includes culturing cells including (i) a translation system; (ii) a reporter mRNA; and (iii) an RNA regulatory sequence in the presence of at least one test compound, wherein the RNA regulatory sequence is not attached to the reporter mRNA. The ability of the test compound to affect the interaction between the RNA regulatory sequence and at least one component of the translation system is sought to be determined. The method further includes monitoring for the effect of the test compound on the this interaction, wherein a compound that modifies this interaction is a drug candidate. | 12-25-2008 |
20080269191 | Compounds for Nonsense Suppression, Use of These Compounds for the Manufacture of a Medicament for Treating Somatic Mutation-Related Diseases - The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA. | 10-30-2008 |