Today, we announced that interim data for a first-in-human, phase 1/2 study of mRNA-3927, an investigational #mRNA #therapy for propionic acidemia (PA), has been published in Nature. This is the first published clinical data utilizing mRNA therapy for intracellular protein replacement.
mRNA-3927 is a novel investigational mRNA therapy that encodes for the PCC enzyme, and has the potential to replace a missing or dysfunctional enzyme.
The premise has always been that an mRNA-based approach to making #medicine could advance at the pace of information – and we believe that our mRNA platform could change how medicines for rare diseases may be delivered in the future.
Read more: https://lnkd.in/erEF2rHG
mRNA-3927 is a novel investigational mRNA therapy that encodes for the PCC enzyme, and has the potential to replace a missing or dysfunctional enzyme.
The premise has always been that an mRNA-based approach to making #medicine could advance at the pace of information – and we believe that our mRNA platform could change how medicines for rare diseases may be delivered in the future.
Read more: https://lnkd.in/erEF2rHG
