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Moderna Says US FDA Selects Rare Genetic Disorder Therapy for Pilot Program
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Moderna Says US FDA Selects Rare Genetic Disorder Therapy for Pilot Program
Jun 6, 2024 5:34 AM

08:13 AM EDT, 06/06/2024 (MT Newswires) -- Moderna ( MRNA ) said Thursday that the US Food and Drug Administration has selected its experimental therapy, mRNA-3705, to treat methylmalonic acidemia, a rare genetic disorder, for a pilot program aimed at supporting clinical trials to advance rare disease therapeutics.

"Selection for this program will enable enhanced communication with the US FDA, resulting in acceleration of our development program as we prepare for pivotal study initiation for mRNA-3705 in 2024," said Kyle Holen, Moderna's ( MRNA ) Head of Development, Therapeutics and Oncology.

mRNA-3705 is being investigated in a phase 1/2 study to assess the safety and tolerability of the treatment administered via intravenous infusion in patients one year and older with the condition.

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