Cell therapy road to commercialization stream
Focus session 1
Tackling the key logistical challenges in the commercial delivery of autologous and allogeneic cell therapies

• A view from the trenches: A clinician's perspective on what works - and what doesn't - incell therapy processing and delivery to the patient
  • What should be the key areas of focus for the industry in terms of educating patients and physicians alike as both autologous and allogeneic cell products scale up to commercial levels?
• Case studies followed by audience questions/panel discussion: Comparing and contrasting the post-manufacturing, pre-treatment pathways for autologous and allogeneic cell therapies
  • What are the key steps/technical options for distribution, transportation and storage?
  • How do you coordinate product release for autologous cell therapy products with the clinical site?
  • What are the optimal mechanisms for tracking patient-specific products?
  • Storage/handling of cell therapies at the clinical site: How to regulate this and what are liability issues? How to alleviate them? At what point/under what circumstances does liability shift from company to physician? Which emerging processing technologies can address potential liability issues?
• Questions from the audience & panel discussion: Debating the pros and cons of clinical site vs. factory-based manufacturing options in terms of logistical issues and scaling-up challenges
  

OR | Gene therapy R&D strategies stream
Focus session 2
Cell therapy/gene therapy crossover products: How are leading candidates performing in the clinic and how are safety issues being addressed?

• What preclinical toxicology work do you need to do (in animal models and single cells alike) and what clinical monitoring is required to guard against potential safety issues moving forward?
  • Addressing the risks associated with vector integration: How are the regulators viewing this aspect?
• Case studies: Clinical development updates from a range of novel ex vivo cell products: Reviewing the latest safety/efficacy data and clinical trial designs
  • What are the prospects for the various approaches: How will they realize their therapeutic potential?
  • What are the differentiators between the companies involved in terms of business model? How do they relate to each specific technical/scientific approach?
• Questions from the audience and panel discussion: How commercially viable are these products? What might the commercialization model look like?
  • What is the product - the virus or the cell?
  • Who should release the transplant and who should transfect the cells? What are the pros and cons of centralized versus onsite processing solutions in the context of GMP compliance?

OR | Workshop
(Highly interactive session for a maximum of 25 participants)
Delivering a new business paradigm for cell & gene therapy companies: Weighing up financing models and collaborative strategies for efficient R&D progress

• Case studies from cell and gene therapy companies that have recently secured significant financing or closed partnering deals: What were the secrets of their success?
  • How to secure VC backing in the current environment?
  • Partnering deal
• Short presentation - case study: Capitalizing on opportunities to partner with non-pharma industry companies
• ...followed by panel discussion/audience Q&A
  • What other opportunities are there for cell & gene therapy companies to partner with non-pharma companies (eg. those from devices, imaging, biomaterials sectors)?
    • What are the challenges specific to each in terms of aligning goals and business models and how are they being addressed?
    • How will the general life science/healthcare trend of focusing on biology at the cellular/molecular level drive further collaboration between cell & gene therapy companies and non-traditional partners? How can the cell & gene therapy industry engage these sectors more aggressively?
  • How can cell & gene therapy companies harness alternative revenue streams to support their R&D?
• Short presentation - case study: Making the case for the 'virtual biotech' - is this model the answer for the quick, cost-effective progression of cell & gene therapies to Phase II and beyond?
• ...followed by panel discussion/audience Q&A
  • Examining the arguments of companies that have maintained core competencies in R&D and manufacturing versus those that have pursued an outsourcing-based model: Understanding the key drivers behind the decision in each case
  • Assessing the pros and cons of outsourcing with CROs and CMOs over the short- and long-terms as the services sector surrounding the cell & gene therapy industry continues to mature
    • What will be the repercussions for Western cell & gene therapy companies if the current trend for CRO migration to Asia continues?

Then | Afternoon plenary session
Product characterization and Mechanism of Action of cell & gene therapies: What data do the regulators require in Phase III?

• Short presentation: FDA update on regulatory requirements for the final drug product
  • Use of products of animal origin, shelf life, potency assay
    • What do you do/how do you approach the regulator where there is no tried and tested potency assay for your application?
• ...followed by audience Q&A/panel discussion: How to begin addressing these challenges in earlier phases?
• Short presentation: How are cell/vector tracking technologies improving to help meet MoA data requirements?
• ...followed by audience Q&A/panel discussion
  • What are the pros and cons of the various emerging technologies for biodistribution tracking?
    • How are issues such as the toxicity and potentially short lifespan of 'labels' being addressed by innovative approaches?
  • How can we be more proactive at sharing cell/vector distribution data to save each individual company/organization having to do the same work?

Close of day 2, followed by a cocktail reception in the exhibition area