Moderna shares Phase 1/2 data for mRNA therapy targeted at rare metabolic disorder
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Vaccine developer Moderna (NASDAQ:MRNA) on Friday reported interim Phase 1/2 data for mRNA-3927, its mRNA therapeutic candidate targeted at the rare metabolic disorder propionic acidemia (PA), which affects about one in 100K -150K individuals worldwide.
Citing data from 16 trial participants who received mRNA-3927 doses across five dose cohorts, Moderna (MRNA) said the study drug was generally well-tolerated with no dose-limiting toxicities or study discontinuations.

The global, open-label study is designed to evaluate mRNA-3927 in those aged one year and older with genetically proven PA, a condition characterized by metabolic decompensation events ((MDEs)) and multisystemic complications.
According to Moderna (MRNA), patients who reported MDEs 12 months before dosing indicated a lower incidence or no events of MDE after receiving mRNA-3927.
However, 15 subjects reported treatment-emergent adverse events, and nine had drug-related TEAEs. According to the company, eight patients developed severe adverse events, most of which were linked to PA and unrelated to mRNA-3927.
The study enters the dose-expansion phase to determine the recommended dose for further studies.


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