NasdaqGS:RAREBiotechs
Phase 3 DTX301 Gene Therapy Data Could Be A Game Changer For Ultragenyx Pharmaceutical (RARE)
Ultragenyx Pharmaceutical recently reported that its Phase 3 Enh3ance trial of DTX301, an investigational AAV8 gene therapy for ornithine transcarbamylase deficiency, met its Week 36 primary endpoint with a statistically significant 18% reduction in 24‑hour plasma ammonia versus placebo and an acceptable safety profile.
Beyond ammonia control, DTX301-treated patients in the study were generally able to reduce ammonia scavenger use, modestly increase protein intake, and report improved daily...