Moderna's rare disease candidate selected for US FDA's START program
Moderna (Nasdaq: MRNA) has announced that the US Food and Drug Administration has selected mRNA-3705 for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.
mRNA-3705 is being developed for methylmalonic acidemia (MMA), a rare, inherited metabolic disorder which can lead to toxic acid buildup in the body, causing significant mortality and morbidity.
Currently, there are no approved therapies for the condition, with standard care including dietary and palliative measures. Liver or combined liver and kidney transplants are the only effective treatments available.
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