Program HighlightsDuring the briefing members of Sangamo's management team will discuss recent achievements and objectives for the 2013-2015 time period including:Clinical
- Sangamo plans to present preliminary data in the first half of 2013 from its clinical trials of SB-728-T (SB-728-1101 and SB-728-902 Cohort 5) for the treatment of HIV/AIDS and expects to have the full data set from these trials by the end of 2013. Both studies are designed to maximize the engraftment of T-cells in which both copies of the CCR5 gene have been disrupted making the cells resistant to infection by HIV. This creates a reservoir of cells that cannot be infected by the virus but are available to mount an immune response with the goal of providing a "functional cure" for HIV.
- Sangamo will outline its strategy to enable the potential filing of seven Investigational New Drug (IND) Applications by the end of 2015.
- The Company will introduce a new, highly disruptive " In Vivo Protein Replacement Platform" driven by Sangamo's ZFP nuclease (ZFN) technology which is potentially curative and leverageable across many monogenic diseases, such as hemophilia and LSDs (e.g. Gaucher, Fabry, and Pompe disease) that are currently treated by regular infusions of enzyme replacement therapy (ERT). Sangamo's novel approach uses a naturally highly expressed gene to drive continuous production of active forms of these proteins from the liver such that they are present at stable levels in the bloodstream thus reducing or eliminating the need for ERT.
- The Company will update investors on recent preclinical progress in ZFP Therapeutics programs with the goal of engineering a genetic cure for hemophilia, Huntington's disease and hemoglobinopathies (sickle cell disease and beta-thalassemia) and outline the key next steps and timelines to IND filing.
- Sangamo will provide a near- and mid-term financial overview including the anticipated amount of cash and cash equivalent of $40-$45 million by the end of 2015 assuming completion of milestones associated with existing partnerships but not including any additional funding from new partnerships, research grants or equity financing transactions.