May 6, 2015

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

What's Hot And What's Not In Gene Therapies for Rare Diseases

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

Furthermore, the opportunities, as well as the issues facing commercializing such new therapeutic approaches, are 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.

The panelists also respond to questions from the audience.

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