Positive interim data from the global Phase I trial of single-dose NTLA-2001 demonstrated a mean serum TTR reduction of 93% at the highest dose level of 1.0 mg/kg by day 28.
Positive interim data from the global Phase I trial of single-dose NTLA-2001 demonstrated a mean serum TTR reduction of 93% at the highest dose level of 1.0 mg/kg by day 28.
CRISPR technology. For amyloidosis but the future is bright for many others like HoFH