Preliminary Phase 1/2 Clinical Trial Results for mRNA-3927, a Potential mRNA Treatment for Propionic Acidemia, Featured in Nature Journal

Moderna, a biotechnology company, has reported preliminary results from a phase 1/2 clinical trial for mRNA-3927, an experimental treatment for propionic acidemia (PA), a rare genetic metabolic disorder. The study, published in Nature, showed that mRNA-3927 was well-tolerated with few side effects and demonstrated a reduction in the frequency of metabolic decompensation events (MDEs) in patients. Kyle Holen, Moderna's Senior Vice President, highlighted the significance of these findings, especially considering the lack of approved treatments for PA.
The trial, registered with the identifier NCT04159103, included participants over one year old with genetically confirmed PA. As of May 31, 2023, 16 participants had received mRNA-3927, with 12 continuing into an extension study after completing the trial. Over 340 doses were administered, equating to more than 15 person-years of treatment. The therapy was found to be safe, with no dose-limiting toxicities observed, and a 70% reduction in the risk of MDEs during the treatment period.
PA is a serious condition that affects approximately one in every 100,000 to 150,000 people globally, caused by mutations in the PCCA and PCCB genes, leading to a deficiency in the propionyl-CoA carboxylase enzyme. This deficiency results in the accumulation of toxic metabolites, causing MDEs and a range of multisystemic complications, including neurological issues and heart problems.
mRNA-3927 is an innovative mRNA therapy delivered intravenously and encapsulated in lipid nanoparticles. It encodes for the PCCA and PCCB subunits to restore the functionality of the deficient PCC enzyme in the liver, potentially replacing the missing or nonfunctional enzyme.
Moderna is a pioneer in mRNA medicine, known for its rapid development of medicines, including a highly effective COVID-19 vaccine. The company's mRNA platform is also being used to develop treatments for various diseases, from infectious to autoimmune conditions. Moderna's commitment to mRNA medicines aims to significantly impact human health.

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