PTC Therapeutics
PTC Therapeutics is a biopharmaceutical company known for developing the first approved treatments for Duchenne muscular dystrophy and gene therapies directly administered into the brain. Their product pipeline includes treatments for various diseases such as Huntington's disease, ALS, and SMA.
Gene Therapy Center of Excellence
PTC Therapeutics operates a Gene Therapy Center of Excellence located in Hopewell, New Jersey. This state-of-the-art facility is dedicated to the development, testing, and manufacturing of gene therapies. The center serves as a hub for innovative gene therapy research and development, aiming to advance treatment options for various genetic disorders.
Approved Treatments
PTC Therapeutics has a portfolio of approved products targeting a range of rare diseases. Noteworthy products include TRANSLARNA™ (ataluren), approved in the EU and Brazil for Duchenne muscular dystrophy caused by a nonsense mutation; Evrysdi® (risdiplam), approved for spinal muscular atrophy (SMA) in the US and EU; UPSTAZA™ (eladocagene exuparvovec), approved for aromatic L-amino acid decarboxylase (AADC) deficiency in the EU, Great Britain, and Israel; and TEGSEDI® (inotersen), approved for polyneuropathy of hereditary transthyretin amyloidosis (hATTR) in adults in the US, Canada, and the EU.
Innovative Pipeline
PTC Therapeutics has a robust product pipeline aimed at developing treatments for diseases like Huntington's disease, amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA). The development programs include a focus on small molecule splicing modifiers and investigational treatments such as TRANSLARNA™ (ataluren) in the US. The company also focuses on treatments targeting splicing modulation to regulate protein production.
Notable Products
Key products from PTC Therapeutics include Emflaza® (deflazacort) for Duchenne muscular dystrophy, vatiquinone for Friedreich ataxia, and WAYLIVRA® (volanesorsen) for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL). Many of these products have received orphan drug designations, highlighting their critical importance in treating rare diseases.
Research and Development Focus
PTC Therapeutics is actively engaged in research targeting ferroptosis and inflammation. The company aims to address oxidative stress and inflammation pathways to offer new treatments for central nervous system (CNS) diseases. Their focused research platforms seek to bring innovative solutions to patients with unmet medical needs.