Accelerating Cell & Gene Therapeutic Success Through Healthcare, Government and Patient Involvement

06 Sep 2023
Stream 3


11:00am Chair Introduction 

Chairperson: George Eastwood, Board Chair, Interim Executive Director, Emily Whitehead Foundation


11:05am Presentation: Opie Jones Foundation: Our Family’s Journey Battling Infant Leukemia 

  • Opie’s story  
  • Our experience receiving a CAR-T therapy  
  • The creation of the Opie Jones Foundation: our objectives, initiatives, and information on how you can get involved 

Lucy Ellerker-Jones, Founder, Opie Jones Foundation 


11:20am Presentation: Bringing Innovation and Regulation together

  • Swissmedic Innovation Office:
    • Mission, Role and Responsibilities
    • Presence and visibility near the innovation, promote Innovation
    • Early dialogue and reduce barrier between Regulator and Stakeholders
  • Swissmedic’s Competence Center Advanced Therapy Medicinal Products (ATMP) :
    • Advanced Therapy Medicinal Products as Pilot Project to develop the Innovation Office
    • Division Advanced Therapy Medicinal Products Specificities
    • ATMP Risk Based Approach –accelerate and facilitate the access of innovative therapies to the patients

Julia Djonova, Head of the Advanced Therapy Medicinal Products, Swissmedic 


11:35am Presentation: Gene therapies: Are the European Healthcare Systems Ready?

  • Characteristics of Gene Therapies and the European access environment  
  • Impact of the EU pharmaceutical legislative review on the ATMP Sector 

Thomas Bols, Head of Government Affairs and Patient Advocacy, EMEA, PTC Therapeutics   


11:50am Presentation: Cell And Gene Therapy For Rare Diseases, Achieving it’s Potential?

  • Huge preclinical hope and progress in all areas of development 
  • Slowest progress though is in clinical translation 
  • Some areas remain poorly understood (eg immunogenicity) 
  • Regulatory challenges and funding models are current greatest obstacles in bringing therapies to patients 

Simon Jones, Professor of Paediatrics and Translational Medicine, University of Manchester


12:05pm Closing Panel with Q&A 

With all session participants joined by:

George Eastwood, Board Chair, Interim Executive Director, Emily Whitehead Foundation




George Eastwood
Board Chair, Interim Executive Director
Emily Whitehead Foundation
Julia Djonova
Head of the Advanced Therapy Medicinal Products
Thomas Bols
Head of Government Affairs and Patient Advocacy, EMEA
PTC Therapeutics
Simon Jones
Professor of Pediatrics and Translational Medicine
University of Manchester