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Sangamo BioSciences Reports Second Quarter 2016 Financial Results

Dr. Macrae continued, "In my first few months I have been reviewing programs and working with the team to understand how best to use our resources to position the Company for success. As part of this process, we are re-prioritizing some of our efforts. In 2016, our intention remains to file an investigational new drug (IND) application for our hemophilia A program which uses an adeno-associated virus (AAV) cDNA gene therapy approach and to begin a Phase 1/2 clinical trial of our in vivo genome editing program in hemophilia B. However, the initiation of our Phase 1/2 trials in MPS I and MPS II is delayed until 2017 and we expect that the timing of milestones related to some of our other programs will change as we refocus our efforts.

"I am confident that this review and future focusing of our efforts will be beneficial to the efficiency and performance of the organization in the long run. I am pleased to be leading Sangamo to a new stage in its development and excited to be a part of building a world class organization capable of developing innovative medicines for patients and creating value for its shareholders."

Summary of Therapeutic Development Program Updates
Sangamo has eight programs in therapeutic development in addition to ongoing clinical trials to evaluate its ZFN-mediated genome editing approach for HIV/AIDS in T-cells as well as hematopoietic stem and progenitor cells (HSPCs). As part of its review and evaluation process the Company is prioritizing efforts to enable development of its therapeutics and execution of it trials to the highest standards.

The current expected status of Sangamo's programs is summarized below. Additional detail will be provided during this afternoon's call.

  • Sangamo expects to file an investigational new drug (IND) application for its AAV cDNA human Factor 8 (hF8) gene therapy program for the treatment of hemophilia A and to initiate the Phase 1/2 clinical trial of its in vivo genome editing approach for hemophilia B in 2016.
  • The Company's proposed Phase 1/2 clinical trials for both Mucopolysaccharidosis Type I (MPS I) and MPS II are expected to begin in 2017 after Sangamo completes certain in vitro preclinical studies and discussions with the U.S. Food and Drug Administration (FDA).
  • IND applications for Sangamo's collaborative programs with Biogen in beta-thalassemia and sickle cell disease are anticipated to be filed in 2017 after some additional preclinical work to optimize the programs. Sangamo will provide more information as it becomes available.
  • Sangamo is evaluating the preclinical development plans for its in vivo genome editing approach for Fabry disease and Gaucher disease to inform the optimal path forward to IND application.
  • The Company expects to present data in 2017 from its ongoing Phase 2 clinical trial in T-cells (SB-728-1101 Cohort 3*) and investigator-sponsored Phase 1/2 clinical study (SB-728mR-HSPC) in HSPCs, both of which are designed to evaluate Sangamo's ZFN-mediated genome-editing approach for HIV/AIDS.

Recent Events

  • Appointment of Sandy Macrae as new president and CEO. In June, Sangamo announced the appointment of Alexander 'Sandy' Macrae, M.B., Ch.B., Ph.D., MRCP, as president and chief executive officer to succeed founder and former president and CEO, Edward Lanphier. Dr. Macrae, a physician scientist, joins Sangamo with deep experience in clinical development and global business strategy, most recently serving as global medical officer of Takeda Pharmaceuticals. Mr. Lanphier stepped down from his chief executive leadership role and, following the Company's 2016 annual meeting of stockholders on June 14, 2016, assumed chairmanship of Sangamo's Board of Directors.
  • Announcement of new gene therapy clinical development program for the treatment of hemophilia A. Following the presentation of preclinical data at the World Federation of Hemophilia 2016 World Congress demonstrating supraphysiological levels of human factor VIII (hFVIII) expression from Sangamo's new proprietary AAV cDNA therapeutic, the Company announced that it is advancing the clinical development of this AAV cDNA approach (SB-525) for the treatment of hemophilia A. Sangamo also announced the goal of filing an IND application with the FDA in 2016 and, pending FDA clearance, initiating a Phase 1/2 clinical trial in adults with hemophilia A.
  • Presentation of new data and recent developments from multiple research and ZFP Therapeutic® programs at 2016 Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT). At the 2016 ASGCT Annual Meeting, Sangamo presented new preclinical data from its proprietary ZFN-mediated in vivo genome editing approach...

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