The promise of gene therapy has rare disease patients not only contemplating the potential of new treatments, but ones that can free them from chronic therapies and potentially provide cures. Sangamo Therapeutics, long pursuing its proprietary gene editing technology, is suddenly moving into the clinic with four experimental therapies including treatments for hemophilia A, hemophilia B, MPS I, and MPS II. The company is currently enrolling three trials and expects to begin enrollment on a fourth trial later this month. We spoke to Sandy Macrae, President and CEO of Sangamo, about the diseases it is targeting, the company’s unique approach to gene therapy, and it’s strategy for moving their therapies through clinical development and to the market.
RARECast is a weekly series by Daniel S. Levine. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013, which produces The Bio Report and RARECast podcasts. Read his full bio here.