Sangamo's Fabry Gene Therapy May Be A STAAR In The Making
Looks To Challenge Enzyme Replacement Therapy Biweekly Infusions
The US biotech has presented interesting preliminary data from the first four patients treated in the Phase I/II STAAR study evaluating isaralgagene civaparvovec for Fabry.
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Avrobio is sidestepping market and regulatory issues in Fabry disease by suspending development of AVR-RD-01, but says it remains focused on advancing treatment of lysosomal storage disorders with gene therapies.
The prospects of lucerastat becoming Idorsia's second drug to be filed with regulators, after the insomnia treatment daridorexant, look slim after a Phase III fail for the Fabry disease therapy.
Shares in Protalix sank more than 30% after the company received a complete response letter from US regulators for pegunigalsidase alfa for Fabry disease. No inspection of the company's manufacturing plant in Israel appears to be the main sticking point.