Gene therapy can revolutionize how we treat patients with rare diseases. As we work to discover and develop therapeutics for patients living with genetic disorders, PTC Chief Scientific Officer, Mark Pykett, explains our strategy and goals for gene therapy.

“PTC is a global company with a commitment spanning more than two decades to patients with rare diseases with high unmet medical need,” explains Pykett. “We focus on neurological and neuromuscular therapeutic areas, along with niche oncology programs. We currently have over 980 employees dedicated to helping patients with poor treatment options, a commercial footprint in 50 countries, with offices in 20 countries, and three commercial products. We have a Biologics License Application under review at the US FDA for our small molecule therapeutic, risdiplam, for spinal muscular atrophy, or SMA, with our commercial partner, Roche. And we have a Marketing Authorization Application under review at the EMA for our gene therapy for AADC deficiency, as well as a robust pipeline of small molecule and gene therapy product candidates.

“So it is a very exciting time for PTC.”