Therapeutic Areas
PTC discovers and develops small-molecule compounds designed to alter post-transcriptional processes and modulate the utilization of messenger RNA, or mRNA, a key intermediate in protein production. Post-transcriptional control processes are of central importance to the regulation of the rate and timing of cellular protein production with the potential to treat a broad range of diseases. PTC has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes to systematically pursue this new approach to drug discovery. PTC's current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology and infectious diseases.
To learn more about PTC's scientific approach please see: Scientific Background and Focus on RNA.
 PROGRAM |
LEAD DISCOVERY / OPTIMIZATION |
PRECLINICAL |
PHASE 1 |
PHASE 2 |
PIVOTAL |
| GENETIC DISORDERS | |||||
| ataluren |  Nonsense mutation (nm) Duchenne/Becker muscular dystrophy (DBMD) |
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| Nonsense mutation (nm) cystic fibrosis
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| Spinal Muscular Atrophy | |
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| DBMD - SERCA2a | |
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| DBMD – Utrophin | |
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| ONCOLOGY | |||||
| PTC299 |  Metastatic breast cancer |
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| Solid tumors |
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| Pediatric brain tumors |
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| Bmi-1 | |
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| INFECTIOUS DISEASES | |||||
| PTC725 |  Hepatitis C |
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| Antibacterial program | |
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| Antiviral programs | |
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| OTHER THERAPEUTIC AREAS | |||||
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