PTC Therapeutics Acquires ST-920 Fabry Disease Program
PTC Therapeutics is acquiring the BLA-stage ST-920 Fabry disease program for $111M upfront and up to $100M in contingent milestone payments
Planned acquisition leverages existing regulatory and commercial infrastructure and leadership's experience in Fabry therapy commercialization — — ST-920 is a one-time administered AAV gene therapy for the enzyme deficient in Fabry disease with demonstrated long-term clinical benefits and safety profile — — BLA submission expected to be completed in Q4 2026 with potential for commercial launch in 2027 — — PTC will host a conference call today, Aug.
12, at 5 p.m.
ET — WARREN, N.J., Aug.
12, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT ) today announced that it was selected as the winning bidder to acquire ST-920 — a BLA-stage one-time administered AAV gene therapy for Fabry disease — from Sangamo Therapeutics in a competitive bankruptcy auction.
The terms include $111 million upfront and up to $100 million in contingent milestone payments based on certain regulatory approvals.
A rolling BLA submission to FDA for accelerated approval of ST-920 is expected to be completed in Q4 2026.
The BLA is based on evidence of meaningful favorable clinical effect on renal function and safety and tolerability profile over 52 weeks in the Phase 1/2 STAAR study.