PTC Therapeutics won rights to Sangamo’s Fabry gene therapy program ST-920 (isaralgagene civaparvovec) in a competitive bankruptcy auction, outbidding Astellas and TerSera Therapeutics. The agreement provides Sangamo $111 million upfront plus an additional $100 million tied to regulatory milestones. ST-920 is a one-time AAV gene therapy intended to restore alpha-galactosidase A production, targeting the lysosomal enzyme deficiency that drives Fabry disease pathology. The Phase I/II STAAR study evaluated patients transitioning off enzyme replacement therapy after treatment, as described in the coverage. For rare-disease investors and developers, the transaction is notable for turning a distressed asset into a near-term revenue opportunity without requiring PTC to build a new commercial platform from scratch.
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