PTC Therapeutics (NASDAQ:PTCT) has a mission to "leverage our knowledge of RNA biology to bring novel therapeutics to patients affected by rare and neglected disorders." The science at PTC is focused on discovering drugs that address so-called "nonsense" genetic mutations and specifically "premature termination codon mutations." These are defects in the DNA sequence in which an amino acid code on a gene is substituted by a stop codon, which halts the sequence and prevents production of a fully functioning protein. PTC mutations are the underlying cause for 10% of all human genetic disease. About 1800 inherited human diseases are caused by nonsense mutations; no treatments are currently available so the unmet medical need is huge.
The PTCT pipeline...
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