PT

PTC Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for patients with rare disorders. The company utilizes its proprietary technologies to target RNA biology and gene therapy, leading to a diverse pipeline of treatments for genetic diseases. Their portfolio includes approved products for Duchenne muscular dystrophy (DMD) and Spinal Muscular Atrophy (SMA), the latter developed in collaboration with Roche and Genentech. PTC is recognized for its expertise in small molecule splicing modifiers and gene therapy delivery systems, aiming to address high unmet medical needs in neurology, oncology, and metabolic diseases.