Moderna's mRNA-3927 therapy for propionic acidemia shows 70% reduction in metabolic events and is well-tolerated in phase 1/2 study.

Moderna has published interim data for its first-in-human phase 1/2 study of mRNA-3927, an investigational mRNA therapy for propionic acidemia (PA), in Nature. The therapy showed a 70% reduction in metabolic decompensation events and was well-tolerated in participants aged one year and older with genetically confirmed PA. The ongoing global Phase 1/2 clinical trial aims to assess safety, pharmacodynamics, and pharmacokinetics of mRNA-3927.

April 03, 2024
7 Articles

Further Reading