Moderna's mRNA Therapy for Propionic Acidemia Shows Promise in Early-Stage Trial

Moderna, Inc. has announced the publication of interim data from a phase 1/2 study evaluating mRNA-3927, an investigational therapy for propionic acidemia (PA), in the scientific journal Nature. This marks the first time clinical data has been published on an mRNA therapy for intracellular protein replacement, offering potential hope for patients with PA, a rare and potentially debilitating metabolic disorder.

The ongoing multicenter study aims to assess the safety, efficacy, and dosing of mRNA-3927 in individuals with genetically confirmed PA. With no current therapies addressing the root cause of PA, mRNA-3927's development is a significant advancement. As of May 31, 2023, the trial had administered over 340 doses to 16 participants, with a positive safety profile and no dose-limiting toxicities observed.

Importantly, the initial findings indicate a reduction in metabolic decompensation events (MDEs) among participants, with a 70% relative risk reduction reported during the study's treatment period. This data supports the potential of mRNA-3927 to provide clinical benefits for PA patients, addressing a significant unmet medical need.

Moderna plans to further investigate mRNA-3927 in a dose expansion phase of the trial, aiming to gather more comprehensive data on its efficacy, safety, and pharmacodynamics. The therapy encodes for proteins essential in mitigating PA's effects, showcasing the broader capabilities of mRNA technology in treating rare diseases.