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April 30 – May 2, 2018

Boston, MA
The Countdown Is On – 1 Week To Go!

Day One
Tuesday, May 1, 2018

Day Two
Wednesday May 2, 2018

Chair’s Opening Remarks

Discussing Pricing, Reimbursement & Market Access Considerations

Establishing Value for Gene Therapy Products by Implementing Effective Reimbursement Strategies

  • Sven Kili VP, Head of Cell & Gene Therapy Development, GSK


  • Considering the patient and carers with broader implications, not just the therapy in reimbursement planning
  • Demonstrating durability through factors like long-term efficacy and safety, which are primary differentiators from current treatment options
  • Investigating new reimbursement models for gene therapy – strengths and weaknesses
  • Learning first hand from experiences in pricing Strimvelis in the European landscape and lessons that can be taken into the US environment

Evaluating Potential Pricing Strategies for Gene Therapies


  • Understanding the relative advantages of up-front payments versus performance over time
  • Striking the optimum balance between access and affordability
  • Investigating how to best compare the cost of treatment with current therapy
  • Evaluating what a patient costs to the health system and factoring this into pricing and value assessments

Panel Discussion: Global Commercialization Considerations


  • Evaluating the challenge of multiple healthcare systems globally – how can pricing and reimbursement structures adapt and change to suit each geo?
  • Investigating how follow-ups can adapt to handle the unexpected: patients who die of natural causes, move or change insurer
  • Learning from the reimbursement of cell and gene therapy products in the US and Europe to inform future strategies

Morning Refreshments & Networking

Enhancing the Efficacy & Safety of Gene Therapy Vectors

The NAV Platform of AAV Vectors for Gene Therapy: Current Applications & Beyond


  • Case studies illustrating clinical applications with the current vector technology platform
  • Critically evaluating current vector technology – what are the key limitations and where are the greatest opportunities for enhancement?
  • Understanding the opportunities enhanced vector technologies will open up

Establishing Robust Approaches to Quantify AAV Vectors and Build a Robust Path to the Clinic


  • Contrasting multiple approaches (qPCR, ddPCR, capsid ELISA) used to characterise and determine vector titer
  • Reflecting on the down-stream effects of robust vector characterization
  • Why is this so important? Understanding the influence of vector titering in deciding on initial clinical starting dose

Lunch & Networking

Putting the Patients First

The Patient Perspective – Understanding Common Misconceptions & Frequently Asked Questions About Gene Therapy


  • Understanding how patients really view the gene therapy space and how organizations can use this information to engage with them more effectively
  • Cutting through the hype – evaluating strategies to inform patients of the reality of inclusion in gene therapy trials
  • Discussing examples of patient misconceptions and questions about the field

Using Feedback from Patient Groups to Shape Gene Therapy Protocols

  • Sharon Hesterlee Research Project Lead, Gene Therapy, Bamboo Therapeutics, A Pfizer Company


  • Patients and patient organizations are a rich source of real world information
  • Patient preferences are increasingly recognized and valued by regulators
  • Gene therapy creates unique benefit/risk situations where patient preference information can inform protocol design

Gene Therapy Clinical Trials: Can They be Compelling for Patients/Families, Investigators and Health Authorities?


  • Understanding the key constituencies of the clinical equation at every level – how and why are gene therapy studies different?
  • Educating patients and families of the realities and risks associated with clinical trials
  • Addressing key health authority questions: are they compatible with relevant clinical programs in rare disorders?

Afternoon Refreshments & Networking

Advancing Gene Therapies to Patients by Forging Mutually Beneficial Alliances


  • Sharing how internal and external capabilities and expertise can contribute to effective clinical development in the gene therapy space
  • Identifying key companies and organizations to collaborate with to advance the development of gene therapies
  • How can organisations in the field collaborate more effectively to bring therapies to patients more rapidly?

Panel Discussion: The Investor’s Perspective – Evaluating How VCs View the Gene Therapy Field


  • Understanding the gene therapy investment landcape – what are the characteristics and considerations unique to this field?
  • To what extent are new investors looking to dilute out the founders?
  • How do VCs view indications with very small patient populations?
  • What are investors looking for in companies in this space?
  • How flexible are investors in defining investment opportunities?
  • Investigating the barriers to entry in the gene therapy field

Chair’s Closing Remarks