Illustration credit: A.E. Kieren
Of all the technologies revolutionizing circa-2016 healthcare, few deserve the tag of “disruptive” as richly as gene therapy does. By treating previously incurable conditions with a single infusion of healthy genes—yes, that grossly oversimplifies it, but stick with us here—gene therapy could well transform the treatment matrix for any number of diseases.
As one of the few organizations that can handle the intense technical demands associated with the production of gene therapies, uniQure has positioned itself as both an A-list partner for companies like Bristol-Myers Squibb as well as a leader in its own right. “Being first with so many things—manufacturing, regulatory approval, pricing—we are the ones who have to blaze the trail,” Aldag notes.
And all of that, somehow, pales beside what could come next, according to Aldag. “We will be able to control genes—switch them on and off as needed. And we'll treat heart failure patients, proving that gene therapy is not only good for rare inherited diseases but also for large acquired ones.”