Sangamo is a clinical stage biotechnology company focused on genomic medicines with the potential to transform patients’ lives using platform technologies in genome editing (both ex vivo cell therapy and in vivo), gene therapy and gene regulation. The Company is focused on three therapeutic areas: inherited metabolic diseases (IMDs), central nervous system (CNS) diseases and inflammatory and autoimmune diseases. Sangamo is a leader in the development of a proprietary technology platform that enables specific regulation of gene expression and gene modification. Sangamo is developing its lead gene therapy asset, SB-525, with Pfizer for hem A which is currently being evaluated in a Phase I/II trial. On April 2, 2019, Sangamo announced interim Phase I/II SB-525 data in hem A from (n=8) patients in the first four dose cohorts of the trial. Notably, the two patients in the highest dose cohort (3e13 vg/kg) reached FVIII levels of 140% and 94% of normal (as measured by one-stage clotting assay) and 93% and 65% (as measured by chromogenic assay) @ week 6 and had not required factor replacement therapy or experience a bleeding event @ 6 – 12 weeks follow-up. Sangamo also announced that it has begun expanding the 3e13 vg/kg dose cohort which could enroll up to five more patients. The Company’s second gene therapy candidate is ST-920 for Fabry disease, which is expected to enter clinical trials in 2019