In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In ran­dom­ized clin­i­cal tri­al da­ta from a small num­ber of pa­tients, Dutch biotech uniQure shared that its gene ther­a­py for Hunt­ing­ton’s dis­ease seems to re­duce the amount of the mu­tant pro­tein re­spon­si­ble for the dis­ease over the course of a year.

In sev­en pa­tients with ear­ly-stage Hunt­ing­ton’s — four who got the treat­ment and three who got a place­bo — mu­tant hunt­ingtin pro­tein lev­els in the cere­brospinal flu­id de­creased by an av­er­age of just over 50% in pa­tients who got the gene ther­a­py com­pared to around a 17% drop in pa­tients who got the place­bo af­ter a year.

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