Sangamo Advances Gene Editing Therapies for Multiple Rare Diseases into the Clinic

The promise of gene editing and 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 a a gene therapy for hemophilia A, and gene editing therapies for 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 editing, and it strategy for moving its therapies through clinical development and to the market.