Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

In first look at mR­NA pro­tein re­place­ment, Mod­er­na touts ear­ly sig­nals of promise for rare meta­bol­ic ther­a­py

LOS AN­GE­LES — Mod­er­na pre­sent­ed ear­ly da­ta on its mR­NA treat­ment for pro­pi­onic acidemia (PA), a rare meta­bol­ic dis­ease where peo­ple can’t break down cer­tain pro­teins and fats, sug­gest­ing pa­tients had few­er se­vere at­tacks char­ac­ter­is­tic of the dis­ease fol­low­ing treat­ment.

It’s the first time Mod­er­na has shared clin­i­cal re­sults of an mR­NA treat­ment de­signed to re­place bro­ken or miss­ing pro­teins in­side a cell. If the tech­nique proves safe and ef­fec­tive in larg­er stud­ies, it could un­lock a swathe or new ap­pli­ca­tions for mR­NA.

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