PTC Therapeutics teamed up with the Alliance for Regenerative Medicine (ARM) to host an interactive panel discussion on cell and gene therapies for rare diseases. This event explored how policy leaders can work together to advance access to cell and gene therapy in Europe. Watch the video!


The growing number of advanced therapy medicinal products (ATMPs) such as cell and gene therapies offers hope to patients with unmet medical need. Some of these therapies are lifechanging, addressing the underlying causes of diseases and replacing lifelong chronic treatment with a single administration or a few injections – a tremendous benefit to patients as well as overburdened healthcare systems!

Much has been accomplished to accelerate cell and gene therapies’ regulatory review and approval. Yet, bringing an ATMP to patients is still quite challenging. Key challenges include:

  • Limitations in running randomized controlled trials
  • Reliance on real world evidence (RWE)
  • Need for highly specialized treatment centers
  • Cost

A better understanding and finding solutions will be crucial to addressing the bottlenecks and adapting the regulatory and access frameworks to advance the treatment of patients with these technologies.

This nteractive panel features experts debating the challenges and future perspectives in creating the necessary conditions to advance the promise of AMTPs.


  • Prof. Guido Rasi, Former Executive Director at the European Medicines Agency
  • Dolors Montserrat, Member of the European Parliament


  • Avril Daly, CEO of Retina International and board member of EURORDIS
  • Flora Giorgio, European Commission, Dg Sante
  • Paige Bischoff, Senior Vice President, Global Public Affairs, Alliance for Regenerative Medicine
  • Thomas Bols, Head of Government Affairs and Public Policy, EMEA & APAC, PTC Therapeutics


Petra Wilson, Ph.D., Managing Director Health Connect Partners & Senior Advisor FTI Consulting