15 Key questions to be debated

1. How are Provenge and other commercial cell/gene therapy and tissue-engineered products being assessed for reimbursement, and what lessons are there for developers of future cell & gene therapy products?
2. How to realize the commercial potential of niche indications and orphan diseases in the context of the evolving pricing and reimbursement environment?
3. Is the VC model really broken in the cell & gene therapy space and, if so, what are the alternatives?
4. Comparing and contrasting the post-manufacturing, pre-treatment pathways for autologous and allogeneic cell therapies: What are the key steps/technical options for product testing, distribution, transportation and storage?
5. Where do the embryonic and induced pluripotent stem cell fields stand today, and what is the real therapeutic potential?
6. How are regulatory pathways developing in key Asian nations including Japan, South Korea and Taiwan?
7. Reviewing recent deals between big pharma/big biotech and cell & gene therapy companies: What data/business model aspects convinced the big pharma/big biotech company to get involved in each example?
8. How are alternative public (eg. State/Government funding for stem cell R&D, NIH grants, Tax Credits) and private (eg. Foundation funding) financing opportunities and initiatives evolving to meet the challenge of delivering approved cell & gene therapy products to patients?
9. Ex vivo cell therapy: How are leading candidates performing in the clinic and how are safety issues being addressed?
10. What opportunities are out there for cell & gene therapy companies to partner with non-pharma companies (eg. those from devices, imaging, biomaterials sectors)?
11. What do you do/how do you approach the regulator where there is no tried and tested potency assay for your application?
12. What insights regarding the selection of pivotal trial endpoints can be drawn from past trials in both cell and gene therapy?
13. When/where/how should automation technology be applied to minimize costs and improve quality/regulatory compliance?
14. Addressing scale-up issues with tissue/cell therapy-device combination products - when to engage a device specialist and when to develop your own?
15. 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?
    

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