PTC Therapeutics Inc. (PTC) is a biopharmaceutical company focused on discovering, developing, and commercializing treatments for rare genetic diseases. Its marketed portfolio includes Translarna (ataluren) and Emflaza (deflazacort) for Duchenne muscular dystrophy (DMD), and Evrysdi (risdiplam) for spinal muscular atrophy (SMA) in pediatric and adult patients. PTC also provides Upstaza, also known as Kebilidi (eladocagene exuparvovec / eladocagene exuparvovec-tneq), for Aromatic L-Amino Decarboxylase (AADC) deficiency. In addition, it offers Tegsedi (inotersen) and Waylivra (volanesorsen) to treat stage 1 or stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis (hATTR). Waylivra is also approved in Brazil for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL).

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PTC has made significant advances in its R&D portfolio, including achieving positive results from the Phase 2 PIVOT-HD study of votoplam in individuals living with Huntington’s Disease and progressing several early-stage programs. The company's R&D efforts are focused on two scientific platforms: RNA splicing, inflammation and ferroptosis. These platforms are aimed at providing access to best-in-class treatments for patients with unmet medical needs. The company continues to invest in its pre-clinical product pipeline and is committed to advancing its early-stage R&D programs. The R&D units of PTC are focused on RNA splicing, inflammation and ferroptosis platforms. These units are responsible for managing the company's R&D activities, including drug discovery efforts, conducting preclinical studies and clinical trials, and manufacturing development efforts. The units also handle activities related to regulatory filings and the advancement of preclinical programs. In FY2025, the company spent US$455.249 million on its R&D activities, which as a percentage of revenue stood at 26.3% and declined 4.5% YoY. As of January 2026, PTC's patent portfolio included a total of 106 active US patents and 53 pending US non-provisional patent applications.