StocksMedium•4 November 2026•
1 min read
Ultragenyx Discontinues Two Gene Therapy Programs Following Trial Failures
Key Facts
1Ultragenyx announced the discontinuation of its GTX-102 program for Angelman syndrome and UX701 program for Wilson disease.
2The decision to terminate the programs follows unsuccessful clinical trial outcomes.
Ultragenyx Pharmaceutical announced the discontinuation of its GTX-102 program for Angelman syndrome and UX701 program for Wilson disease. According to Benzinga, the decision to terminate these developments follows unsuccessful clinical trial outcomes, with GTX-102 failing to demonstrate efficacy in its Phase III study.
RARE closed at $16.63 on November 3, 2026, as the company moves to reduce R&D expenses by more than 50% by 2027. The firm stated these measures include a 20% headcount reduction, while it reaffirmed its 2026 revenue guidance of $730 million to $760 million.