Print Programme


Clinical Trial and Commercialisation Workshop
Tuesday 22 October 2019
Room 113
Organisers: Alessandro Aiuti (TIGET) - Robin Ali (UC, London) - Federico Mingozzi (Spark Therapeutics)
09:30-11:00 Planning and running a clinical trial

INV001: Gemma Jones 
UCL Clinical Trials Centre, London
General considerations for setting up a clinical trial

Patient identification & selection

INV002: Alessandro Aiuti
SR Tiget, Milan
Newborn screening programs for rare monogenic disorders:
Impact on early detection and treatment with ATMP

INV003 Ron Philip
Spark Therapeutics, Philadelphia, PA
Genotyping for inherited retinal disorders

INV004: Alden Ladd
bleubirdbio, Cambridge, MA
Towards commercial CAR-T cell therapies: A step-wise approach to CAR T cell product characterisation
11:00-11:30 COFFEE BREAK
Manufacturing and distribution of gene and cell products

INV005: Eduard Ayuso
Université de Nantes
AAV manufacturing challenges

INV006: Reed Clark
Sarepta Therapeutics, Cambridge, MA
AAV manufacturing: Key considerations and challenges


Gene therapy and commercialisation challenges

INV007: Rocio Martin Hoyos
Audentes Therapeutics, San Francisco, CA
Commercialisation of gene therapies in ultra rare diseases

INV008: Gabor Veres
BioMarin Pharmaceutical, Novato, CA
Development of valoctocogene roxaparvovec

INV009: Patrizio Armeni 
Bocconi University, Milan
Fast tracking in Europe; Challenges of commercialisation

13:30- 14:30 LUNCH
14:30-16:30 Regulatory and reimbursement challenges

INV010: Sol Ruiz
Spanish Medicine Agency, Madrid
How can we lower the regulatory burden?
Challenges for the future / roadmap for the next 5 years

NV011: Giorgio Iotti
Chiesi, Parma
Key considerations in clinical development and commercialisation of advanced therapy medicinal products

INV012: Sam Parker
Lysogene, Paris
Measures of outcome in neurodegenerative gene therapy trials

INV013: Simone Boselli 
Eurordis, Brussels
Role of patient organisation in the development

INV014: Diego Ardigo
Chiesi, Parma
Mapping of all steps / Orphan drug development guidebook