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 white paper, based on a web panel held in May of 2015, addresses cutting-edge advances in genetic therapies likely to provide game-changing treatments, if not cures, for patients with rare inherited disorders. 

Discussion topics covered 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.

Furthermore, the opportunities, as well as the issues facing commercializing such new therapeutic approaches, were 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 white paper also includes questions from the audience and the answers from the panelists.

the white paper