Phase 1/2 Interim Data on Moderna’s mRNA-3927, an Investigational mRNA Therapy for Propionic Acidemia, Published in Nature - Seite 2

Additional participants are now being enrolled into the study as part of a dose expansion phase to allow for further characterization of the efficacy, safety, and pharmacodynamic activity of mRNA-3927.

About Propionic Acidemia

Propionic acidemia is a rare, serious, inherited metabolic disorder with significant morbidity and mortality, affecting one in 100,000-150,000 individuals worldwide. PA is caused by pathogenic variants in the propionyl-coenzyme A carboxylase (PCC) α or β subunits (PCCA and PCCB genes, respectively), leading to PCC deficiency and subsequent accumulation of toxic metabolites. PA is characterized by recurrent life-threatening MDEs and multisystemic complications. Some of the most common multisystemic complications include neurological manifestations, cardiomyopathy, arrhythmia, growth retardation, recurrent pancreatitis, bone marrow suppression, and predisposition to infection. Long-term, insults by toxic metabolites cause multiorgan complications and are correlated with recurrent MDEs.

Currently, there are no effective therapies for PA that target the underlying root cause of the disease.

About mRNA-3927

mRNA-3927 is a novel, IV-administered, lipid nanoparticle (LNP)-encapsulated dual investigational mRNA therapy that encodes for PCCA and PCCB subunit proteins to restore functional PCC enzyme activity in the liver. By encoding for the PCC enzyme, mRNA-3927 has the potential to replace a missing or dysfunctional enzyme.

About Moderna

Moderna is a leader in the creation of the field of mRNA medicine. Through the advancement of mRNA technology, Moderna is reimagining how medicines are made and transforming how we treat and prevent disease for everyone. By working at the intersection of science, technology and health for more than a decade, the company has developed medicines at unprecedented speed and efficiency, including one of the earliest and most effective COVID-19 vaccines.

Moderna's mRNA platform has enabled the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and autoimmune diseases. With a unique culture and a global team driven by the Moderna values and mindsets to responsibly change the future of human health, Moderna strives to deliver the greatest possible impact to people through mRNA medicines. For more information about Moderna, please visit modernatx.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn.

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