PTC Confident About Commercial Prospects For Gene Therapy Upstaza

One-Time Injection Gets CHMP Nod For AADC Deficiency

PTC CEO Stuart Peltz tells Scrip that cost watchdogs are likely to look favorably on the firm’s transformational gene therapy for a distressing rare brain disorder in children who could not even lift their heads before treatment.

Stu Peltz
CEO Stuart Peltz • Source: PTC

The commercial viability of gene therapies has been in the headlines again, but PTC Therapeutics, Inc. is confident that Upstaza, which has just been recommended for approval by EU regulators for an ultra-rare brain disorder, will prove to be a financial as well as medical success story.

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Gene Therapies

More from Advanced Therapies