- Sangamo Therapeutics Inc SGMO announced preliminary results from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a gene therapy candidate for Fabry disease.
- As of the September 17, 2021 cutoff date, results from the four patients treated in the first two dose cohorts (0.5e13 vg/kg and 1e13 vg/kg) showed that the gene therapy was generally well tolerated.
- All four patients exhibited above normal alpha-galactosidase A (α-Gal A) activity, which was maintained for up to one year for the first patient treated and through 14 weeks for the most recently treated patient.
- 2-fold to 15-fold above mean normal was observed at the last measurement as of the cutoff date.
- Withdrawal from enzyme replacement therapy (ERT) has taken place for one patient. It is planned for the other patient on ERT, based on the stability of their α-Gal A activity following treatment.
- No treatment-related adverse events higher than mild and treatment-related serious adverse events were observed.
- No patients experienced liver enzyme elevations requiring steroid treatment.
- Price Action: SGMO shares gained 19.2% at $10.89 on Thursday.
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