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On November 4, 2021, Sangamo Therapeutics announced that the gene therapy isaralgagene civaparvovec (ST-920) has obtained positive preliminary results in a phase 1/2 clinical trial in patients with Fabry disease
Fabry disease is a lysosomal storage disease caused by mutations in the galactosidase alpha gene (GLA), which leads to the lack of AGA activity and the inability to metabolize glotriose ceramide (Gb3) normally
Isaralgagene civaparvovec is a one-time infusion, liver-targeted gene therapy
▲The mechanism of action of Isaralgagene civaparvovec (picture source: reference [2])
As of September 17, 2021, the results of the first two dose cohorts (0.
In addition, in one patient whose plasma biomarker lyso-Gb3 was significantly increased before treatment, within 10 weeks of treatment with isaralgagene civaparvovec, the biomarker was significantly reduced by about 40%, and the effect was maintained until the 32nd week
▲AGA activity level of 4 patients increased to higher than normal level (picture source: reference [2])
In terms of safety, isaralgagene civaparvovec is usually well tolerated
The evaluation of the third cohort (3^13 vg/kg) of the trial is still in progress
Reference materials:
[1] Sangamo Therapeutics Announces Preliminary Phase 1/2 Data Showing Tolerability and Sustained Elevated AGA Enzyme Activity in Patients With Fabry Disease.
[2] Fabry Disease: Preliminary Data Readout for Isaralgagene Civaparvovec (ST-920).
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