2023 is a meaningful, unique and thrilling year for PTC. It’s the company’s 25th anniversary, and it is poised to be a year of momentum as we anticipate significant trial readouts in multiple disease areas. We’re also working toward our ambitious goal to produce a therapy every two to three years based on our robust and diversified platform. This year, we also look forward to celebrating our enduring legacy and delivering on our promise to create more moments for those who count on us.

What’s so special about 2023? Read below to learn:

PTC’s 25th Anniversary

This year, we celebrate 25 years of translating science and transforming lives. We can’t wait to share our proudest achievements, moments and impact throughout our history with you. Stay tuned throughout the year to learn about the company’s history and the incredible people who made an impact on PTC, celebrate with our growing global community of #OnePTC employees and look ahead to the next 25 years.

Significant Trial Readouts

We’re anticipating data readouts from several clinical trials:

  • MIT-E: Registration-directed trial of vatiquinone for Mitochondrial Disease Associated Seizures (MDAS)
  • MOVE-FA: Registration-directed trial of vatiquinone for Friedreich ataxia (FA)
  • APHENITY: Registration-directed trial of sepiapterin for phenylketonuria (PKU)

Anticipated Treatment Milestones

We plan to submit a Biologics License Application (BLA) to the U.S. FDA in the first half of this year for Upstaza™ (eladocagene exuparvovec), our innovative gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency.

Continued Pipeline Advancement

Our pipeline continues to progress:

Supporting Patient Voices and Experiences

Hope you’re as excited about 2023 as we are!