The complexity of protein production associated with protein disorders, especially from faulty genes, is formidable. But that hasn’t stopped PTC from innovating in the space of RNA targeting for novel medicines.
In 2003, PTC discovered the first novel small molecule capable of suppressing premature termination of translation by allowing the ribosome to selectively read through a premature stop codon in mRNA created by nonsense mutation – i.e. nonsense readthrough. This has led to innovative new therapies for treating the rare disease Duchenne muscular dystrophy.
Watch as PTC Founder and CEO Stuart Peltz, Ph.D. discusses RNA biology in the area of post-transcriptional control (PTC) processes, involving mRNA turnover translation. with Europabio, the European Association for Bioindustries.
EuropaBio has recognized PTC as a company that has contributed to one of the most significant biotechnology advances over the last 25 years.