Web Panel Discussion: What’s Hot & What’s Not in Gene Therapies for Rare Disorders


  • Michael C. Rice, MS, MBA, Principal, Defined Health


  • Stewart Abbot, Ph.D, Executive Director, Integrative, Research at Celgene Cellular, Therapeutics (CCT)
  • Paul Gallagher, MBA, President, Compass Strategic Consulting
  • Matthew Porteus, MD, Associate Professor of Pediatrics, (Cancer Biology), Stanford University

Gene and cell therapy technologies have been among the hottest biotechnology sectors for IPOs and licensing deals. This panel will discuss cutting-edge advances in genetic therapies likely to provide game-changing treatments, if not cures, for patients with rare inherited disorders.

Discussion topics will cover advanced viral vector platforms likely to reach the market in the next couple of years, and what is on the horizon with all the excitement around recent breakthroughs in gene editing.

Furthermore, the opportunity, as well as the issues facing commercializing such new therapeutic approaches, will be debated from the vantage points of gene therapy pioneers, biopharma executives developing business models to deliver therapies to patients, and payer perspectives of how such potentially curative one-time treatments are likely to be reimbursed in the US and other payer systems.

If you are interested in rare disorders and how these recent breakthroughs may redefine how therapies are provided to patients, you will want to hear these perspectives.

Throughout the discussion, the panelists will respond to questions from the audience.