What are the limits of gene therapy?

“There are no currently available FDA-approved gene therapy treatments for overactive bladder,” reads this press release. And that’s true. But is it, like, a problem?

Urovant Sciences believes so, which is why it licensed just such a gene therapy for overactive bladder. It’s called hMaxi-K, and it targets a curiously named gene called slowpoke, which plays a role in smooth muscle function. The treatment’s inventor, a company called Ion Channel Innovations, tried it out in erectile dysfunction and came up with nothing. But a second trial, enrolling 13 women with overactive bladder, showed some promise, according to Urovant, and now the company wants to take it into Phase 2 next year.

STAT’s Adam Feuerstein isn’t buying the idea, writing that “a gene therapy for overactive bladder strikes me as a wasteful and unnecessary scientific pursuit.”

To be fair to Urovant, overactive bladder is a very real condition, and there are patients who are burdened by it despite taking available therapies. But gene therapies, at least the ones we’ve seen so far, are risky and expensive to develop, which is in part why the field has focused on treating the rare and often life-threatening diseases that can command premium pricing. Assuming hMaxi-K works, is overactive bladder the kind of condition the system will pay hundreds of thousands of dollars to treat?