SGMO - Sangamo's gene therapy for Fabry Disease shows safety in interim phase 1/2 trial data

Sangamo Therapeutics (NASDAQ:SGMO) said preliminary data from a phase 1/2 study, dubbed STAAR, of its gene therapy isaralgagene civaparvovec (ST-920) continued to be well tolerated in patients with Fabry disease. Fabry disease is a rare inherited disorder of glycosphingolipid (fat) metabolism due to deficient activity of lysosomal enzyme, alpha-galactosidase A (?-Gal A). The company said the four treated patients continued to exhibit elevated ?-Gal A activity. In the first two dose groups, all four patients showed above normal ?-Gal A activity, ranging from 3-fold to 15-fold above mean normal. Patients in the first dose cohort have maintained elevated activity for one year and are now in the long-term follow-up study. In the third dose group, the fifth patient showed activity within mean normal ?-Gal A levels at week 2 and the sixth patient was recently dosed. The company noted that the therapy was generally well tolerated across three dose cohorts in

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Sangamo's gene therapy for Fabry Disease shows safety in interim phase 1/2 trial data