STAT+: Why a Huntington’s patient isn’t fazed by the waning effect of UniQure’s gene therapy

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.This story first appeared in Adam’s Biotech Scorecard, a STAT newsletter. Sign up here to get it delivered to your inbox. And subscribe to STAT+ to get an unabridged version, with exclusive content. Lauren Holder is an advocate for the Huntington’s disease community who is also living with the early stages of the progressive and fatal neurodegenerative condition. I called her Tuesday to get her reaction to UniQure’s gene therapy clinical update. The UniQure treatment, called AMT-130, slowed the progression of Huntington’s by 44%, based on a four-year analysis of 12 treated patients. Last year, the company reported a three-year update showing a 75% slowing of disease progression. The treatment effect of AMT-130 seems to have waned from year three to four, for reasons that don’t alarm UniQure but that have raised some outside questions about the therapy’s durability. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe