Solid has also pivoted from developing SGT-001 to SGT-003 for treating Duchene muscular dystrophy.
Participants in the AAVIATE trial had up to an 85% decrease in treatment burden 6 months after treatment with RGX-314.
Newborn Cell Therapies Group at Monash University has received a $50,000 grant from Lions Cord Blood foundation.
Among the 11 patients evaluated for efficacy, 9 patients (81.8%) had objective responses at 28 days.
Disease-free survival was 56% in 105 participants over 3 years.