Sangamo BioSciences Gets FDA Approval For Its IND For ZFN-Mediated Genome Editing Treatment Of MPS II

Sangamo BioSciences, Inc.

, the leader in therapeutic genome editing, disclosed Monday that the Food and Drug Administration (FDA) has cleared its Investigational New Drug application (IND) for SB-913. The drug candidate is a zinc finger nuclease (ZFN)-mediated approach designed as a single treatment with the potential to offer a long-lasting therapy for Mucopolysaccharidosis Type II (MPS II, Hunter syndrome). According to the company, the IND is now active and allowed it to commence a Phase 1/2 clinical study to assess the safety, tolerability and potential efficacy in adults with MPS II. Sangamo BioSciences' President and CEO, Sandy Macrae, commented, "Our gene-based approach is designed to give the patient the ability to make their own replacement enzyme and has the potential to provide significant clinical and quality of life advantages over repeatedly administered enzyme replacement therapy, which is the current standard of care for MPS II and a number of other genetic diseases." He continued to say, "Unlike conventional, non-integrating AAV gene therapy that has the potential to 'wash out' over time as the patient's liver cells divide and turn over, SB-913 has the potential to provide a uniquely durable solution through genome editing. Ultimately, our target population will include pediatric patients who can benefit from a more durable solution. We believe that a ZFN-mediated genome editing approach can best serve the broadest group of MPS II patients and their families." Sangamo BioSciences EVP R&D, Geoff Nichol, reacted to say, "Our team has successfully prepared and filed three IND applications in short succession, for hemophilia B, MPS I and now MPS II, meeting our stated goal for filing the third IND application for our IVPRP genome editing approach in the first half of 2016. Our next goal is to continue this momentum and initiate the SB-913-1602 clinical study for MPS II in the second half of 2016. With positive clinical and safety data in adults, our intention with this, and all of our IVPRP programs, is to extend trials into pediatric populations who could benefit most from early intervention with a genome editing approach designed to provide stable expression of therapeutic enzyme throughout the patient's lifetime." The stock shed 4.75 percent on Friday.