Clinical ProgressAMT-130 showed statistically significant and dose-dependent slowing of disease progression and a reduction in neurofilament light chain at 24 months.
Manufacturing ConfidenceThe process for manufacturing is highly comparable to Hemgenix and is done in an FDA-licensed facility, indicating confidence in production capabilities.
Regulatory ApprovalQURE announced they have reached regulatory alignment on a path to accelerated approval with the FDA that will not require them to run an additional study, removing the overhang on the stock for many investors.