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