Cell therapy road to commercialization stream
Focus session 1
Piecing together the technology jigsaw to enable the economically-feasible commercial scale-up of cell therapies

• When should you delay clinical advancement in favour of further process/product development to improve the cost/performance aspect?
  • How do you assess what your product price point might be, and then accurately estimate the improvements that might be made on the unit cost and product performance?
  • What impact might the continued development have both on costs and on potential price?
• Cell therapy company case study: Assembling technology components into a platform - what were the decision-making processes and rationale behind our choices?
  • Which specific components are required to balance the cost of goods/manufacturing vs. product performance equation, and how are we strategizing to optimally access and integrate them?
    • How much to invest in optimizing the process with consideration to CoG?
  • How to optimize strategic decision-making in terms of biomaterials sourcing?
• When/where/how should automation technology be applied to minimize costs and improve quality/regulatory compliance?
• Short presentation(s) followed by panel discussion: Addressing scale-up issues with tissue/cell therapy-device combination products
  • When to engage a device specialist and when to develop your own? Examining the pros and cons of each approach, and the decision-making processes for companies that have chosen either pathway

OR | Gene therapy R&D strategies stream
Focus session 2
Focus on the eye: Comparing and contrasting the scientific approaches and clinical data for innovative gene and RNAi therapies in the ophthalmology area

• Case studies: Clinical development updates in the age related macular degeneration (AMD) area with varying gene and RNAi therapy technologies/methods of delivery
  • Overcoming targeted delivery issues: What specific approach is each company employing and why?
  • How do the clinical development strategies tie in with the company's overall business models?
• Questions for the speakers, followed by panel discussion
  • Comparing and contrasting viral and non-viral delivery platforms for ophthalmology therapeutics - which are achieving the desired safety and efficacy profiles? Where do challenges remain?
  • Examining clinical trial endpoints for gene and RNAi therapies in ophthalmology indications - are they good enough? What do these candidates really need to show in trials to ensure there is going to be a viable product at the end?
  • What is the ideal combination of delivery device and vector for ophthalmology indications?

OR | Workshop
(Highly interactive session for a maximum of 25 participants)
In-depth regulatory workshop

• Addressing specific issues arising from disharmonization between US and European regulations for cell & gene therapies

Close of the Phacilitate Cell & Gene Therapy Forum 2011 followed by buffet lunch in the exhibition area