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A journey with many milestones


  • Upstaza™ (eladocagene exuparvovec) for the treatment of AADC deficiency, the first ever gene therapy directly administered into the brain, was approved in Europe and the UK


  • Evrysdi® (risdiplam) approved in the EU and Japan for patients with spinal muscular atrophy (SMA)
  • Received Gallup’s “Don Clifton Strengths-Based Culture” Award
  • Proof of mechanism established for PTC518 demonstrating broad distribution for Huntington’s disease
  • Expanded partnership with the SMA Foundation to advance drug discovery and development research in regenerative medicine


  • Expanded clinical pipeline to include a treatment for phenylketonuria (PKU)
  • Opened a New Biologics Facility in Hopewell, NJ for gene therapy manufacturing
  • Evrysdi® (risdiplam) approved by the FDA for patients with spinal muscular atrophy (SMA)
  • Initiated trial with emvododstat, a potential oral treatment for COVID-19


  • Expanded platform technologies with the acquisition of a bio-electron transfer platform to address diseases of oxidative stress
  • Announced clinical results for its gene therapy treatment for patients with AADC deficiency that demonstrated sustained improvements
  • Announced Real-World Evidence demonstrating Translarna™ (ataluren) slows disease progression in patients with Duchenne muscular dystrophy


  • Addition of Tegsedi® (inotersen) and Waylivra® (volanesorsen) expands the product portfolio in Latin America
  • Acquired a targeted gene therapy platform targeting rare CNS disorders such as AADC deficiency


  • Acquired Emflaza® (deflazacort) for the treatment of Duchenne muscular dystrophy: The only drug approved in the US for all patients living with Duchenne


  • Translarna™ (ataluren) is approved in the EU: The first drug in the world to be approved for Duchenne muscular dystrophy


  • Successful IPO in the US public markets (NASDAQ: PTCT)


  • PTC and the SMA Foundation partner with Roche to continue the development of a treatment for spinal muscular atrophy (SMA)


  • Awarded grant from the Wellcome Trust that supported research and development of unesbulin


  • Partnered with the SMA Foundation to discover and develop a treatment for spinal muscular atrophy (SMA)


  • Initiated the first clinical trials for PTC124, the compound that would become Translarna™ (ataluren), to target the underlying cause of rare disorders caused by a nonsense mutation


  • PTC founded with the mission to leverage expertise in RNA biology to discover, develop, and commercialize treatments for patients with rare disorders

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