Skip to main content

A journey with many milestones

2023 NASDAQ bell ringing


  • Matthew B. Klein, M.D. appointed Chief Executive Officer
  • Statistically significant results from sepiapterin Phase 3 PKU trial
  • PTC celebrates its 25th anniversary and 10 years as a public company
Brain image


  • 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
Microscope and tubes


  • 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
Lab equipment


  • 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)
Lab equipment


  • 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
Lab equipment


  • 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
Lab equipment


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


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


  • 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)
Researcher working at desk


  • 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)
Microscope lenses


  • 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
Stuart Peltz and Allan Jacobsen, founders of PTC Therapeutics


  • PTC was co-founded by Dr. Stuart Peltz and Dr. Allan Jacobson, with the mission to leverage expertise in RNA biology to discover, develop, and commercialize treatments for patients with rare disorders

We'd love to hear from you