Sangamo Therapeutics Inc (NASDAQ:SGMO) announced updated preliminary results from the Phase 1/2 STAAR study of isaralgagene civaparvovec (ST-920), Fabry disease gene therapy candidate.
- Fabry disease is an inherited disorder that results from the buildup of a particular type of fat, called globotriaosylceramide (Gb3), in the body's cells.
- It is caused by mutations in the galactosidase alpha gene (GLA), resulting in deficient alpha-galactosidase A (α-Gal A) enzyme activity, necessary for metabolizing Gb3.
- Elevated α-Gal A activity was maintained for the four patients treated in the first two dose cohorts (0.5e13 vg/kg and 1e13 vg/kg), ranging from 3-fold to 15-fold above mean normal at last measurement.
- Related: Why Did Sangamo Stock Jump 20% Today?
- For the two patients on enzyme replacement therapy (ERT), α-Gal A activity measured at ERT trough was 15-fold above mean normal at week 52 (Cohort 1) and 10-fold above mean normal at week 25 (Cohort 2).
- For the two ERT pseudo-naïve patients, α-Gal A activity was 3-fold above mean normal at week 52 (Cohort 1) and 4-fold above mean normal at week 40 (Cohort 2).
- The gene therapy candidate was generally well tolerated across three dose cohorts in the five treated patients.
- The sixth patient in the Phase 1/2 STAAR study is the second patient in the third dose cohort (3e13 vg/kg). Sangamo expects to provide updated results from the STAAR study throughout 2022.
- Price Action: SGMO shares closed at $5.72 on Monday.
