Pharmaceutical Executive April 4, 2024
Early results have demonstrated that mRNA-3927 has led to a 70% reduction in the relative risk for metabolic decompensation events in patients with propionic acidemia.
Moderna has announced promising data from its Phase I/II study for mRNA-3927, an investigational mRNA therapy for the potential treatment of propionic acidemia (PA). Currently in progress, the open-label, dose optimization and extension trial is evaluating the safety and efficiency of the medication. So far, the trial has included 16 participants, with over 340 doses of the treatment administered. Additionally, no significant adverse events (AEs) have been reported that were related to mRNA-3927. The study’s biggest finding was that the relative risk for metabolic decompensation events (MDEs) were reduced by 70%.1
“We are excited to...