SGMO - Sangamo gene therapy for Fabry disease shows promise in phase 1/2 trial

Sangamo Therapeutics ( NASDAQ: SGMO ) reported updated preliminary results from a phase 1/2 study of its gene therapy isaralgagene civaparvovec (ST-920) to treat Fabry disease.

Fabry disease is a rare inherited disorder of glycosphingolipid (fat) metabolism due to the absence or deficient activity of the lysosomal enzyme, ?-galactosidase A.

In the study dubbed, STAAR, ST-920 continued to be generally well tolerated, with no treatment-related adverse events above Grade 1 (mild), according to Sangamo.

The five longest treated patients showed elevated alpha-galactosidase A (?-Gal A) activity, ranging from about 3-fold to nearly 17-fold above mean normal, up to 15 months as of the last date of measurement, the company noted in an Aug. 30 press release.

Sangamo added that one patient was withdrawn from enzyme replacement therapy (ERT) and showed significantly elevated levels of ?-Gal A activity at 12 weeks post withdrawal.

The company said that since the cutoff date of Feb. 14, an additional five patients have been dosed and an additional four patients in the dose escalation phase have been withdrawn from ERT.

Sangamo noted that the study has progressed into the dose expansion phase and expects to provide additional results from the trial in H2 2022.

The company is currently planning for a potential phase 3 trial.

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