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Moderna: positive data against propionic acidemia

( - Moderna today presented interim results from the Phase 1/2 clinical trial of mRNA-3927, an investigational mRNA-based therapy for propionic acidemia (PA), at the American Society of Gene + Cell Therapy (ASGCT) 2023 Annual Meeting.

The ongoing global Phase 1/2 clinical trial is an open-label, multi-center study designed to evaluate the safety, pharmacodynamics and pharmacokinetics of mRNA-3927 in participants aged one year and older with genetically confirmed PA.

mRNA-3927 was well tolerated at the doses administered, with encouraging early evidence of dose-dependent pharmacology and potential clinical benefit.

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