Sangamo Biosciences (SGMO) has released an update.
Sangamo Therapeutics, Inc. has released promising preliminary data from its ongoing Phase 1/2 STAAR study of isaralgagene civaparvovec, a gene therapy for Fabry disease. As of September 2023, 24 patients have been treated, showing general tolerability and sustained elevated levels of α-Gal A activity, potentially reducing the need for enzyme replacement therapy (ERT). Moreover, improvements in disease severity and quality of life indices were observed, along with a notable reduction in antibodies associated with ERT. Further dosing and long-term follow-ups are planned, with discussions ongoing with health authorities regarding the path to registration.
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For a comprehensive understanding of the announcement, you can read the full document here.
