Sang­amo team rush­es to de­fend first hu­man gene edit­ing da­ta as MPS II ther­a­py of­fers a hit — and a miss

Sang­amo $SG­MO rolled out its first glimpse at the clin­i­cal ef­fi­ca­cy of their gene edit­ing ther­a­py for Hunter syn­drome (MPS II). And while re­searchers were able to point to some clear ev­i­dence that their ap­proach had an im­pact on a key bio­mark­er for the dis­ease for 2 pa­tients, there was a crit­i­cal lack of proof that it was do­ing specif­i­cal­ly what it was de­signed for.

Un­der­stand­ably, Sang­amo con­cen­trat­ed on uri­nary gly­cosamino­gly­cans (GAGs), high­light­ing big drops for both pa­tients in the sec­ond co­hort of the tiny ex­plorato­ry study. 

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