An­oth­er play­er in the gene ther­a­py field is gun­ning for the Duchenne MD crown — and here’s what they're bet­ting on

Most of you gene ther­a­py en­thu­si­asts read­ing this will al­ready know about one pro­gram out of Na­tion­wide Chil­dren’s Hos­pi­tal that’s at­tract­ed con­sid­er­able at­ten­tion in the in­dus­try. It’s the mi­cro-dy­s­trophin one at Sarep­ta, which has snagged a big spot­light for the ear­ly — and very ex­cit­ing — hu­man da­ta that they’ve been rolling out.

To­day, a dif­fer­ent biotech com­pa­ny you most like­ly haven’t heard near­ly as much about is rolling out a new gene ther­a­py pro­gram for Duchenne mus­cu­lar dy­s­tro­phy — soon to be test­ed in hu­mans — that they be­lieve can leapfrog Sarep­ta straight to pa­tients.

“It’s safer and more ef­fec­tive com­pared to oth­er ap­proach­es, in­clud­ing mi­cro-dy­s­trophin.”

That’s the CEO of Au­dentes talk­ing. Matt Pat­ter­son. He’s the com­pa­ny co-founder who count­ed him­self as em­ploy­ee #1 for the first 6 months, now with a staff of 230 and a mar­ket cap of $1.7 bil­lion. Au­dentes has some ear­ly da­ta due out on their lead gene ther­a­py pro­gram for AT132 in a few weeks.

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