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A journey with many milestones

About Our Founder

Dr. Peltz led PTC Therapeutics as its CEO for 25 years, since its founding in 1998 to March 2023. He pioneered the field of RNA-directed drug development. Under his leadership, PTC grew from a research organization with expertise in the control of RNA processes to a publicly traded, integrated, global biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for the treatment of neurological and neuromuscular disorders, metabolic disorders, and cancer.


  • 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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