The company will trim its headcount to approximately 1,000 employees while shuttering all ongoing studies for the Angelman syndrome treatment GTX-102. Simultaneously, management is pulling the plug on the UX701 program for Wilson disease, citing a failure to reach essential study goals. Beyond personnel and clinical cuts, the firm intends to shrink its physical real estate footprint to lower overhead.

Financial targets for 2027 project research and development spending falling below $350 million, with selling, general and administrative costs capped at $375 million. These austerity measures follow a third-quarter earnings report that showed revenue growth of 8% to $173 million. While the company narrowed its quarterly loss to $118 million—down from $180 million during the same period last year—investors reacted sharply to the news of the narrowed R&D pipeline.