FDA says 'not yet' to Verve liver gene editing therapy IND

Nov. 7, 2022

Boston-based biotech Verve Therapeutics announced this week that the FDA had placed a hold on its IND application for its single-course, in vivo liver gene editing treatment for patients with heterozygous familial hypercholesterolemia (HeFH), VERVE-101 .

As a part of its third-quarter financial results reports, Verve shared that it had been given a hold notice last Friday, Nov 4, 2022..The company says it is expecting an official letter outlining the FDA’s concerns within the next 30 days. 

HeFH is a genetic disorder characterized by dangerously elevated levels of low-density lipoprotein cholesterol, and relatively common. Because it is an autosomal dominant disorder, only one parent needs to have an LDLR gene mutation for it to be expressed in offspring. The drug uses an LNP-mediated delivery to make a single base change at the site of the affected gene. 

Elsewhere, VERVE-101 is being tested in a phase 1 clinical trial in New Zealand and the U.K. The company said that they had completed dosing of the drug in the first dose cohort of the dose-escalation portion of the trial, and was tolerated well in all patients. 

When successful, gene and cell therapy drugs bring hope about a future where certain diseases could potentially be eradicated. But fulfilling this promise requires many hands and brains that the industry is having a hard time finding. Read more about this in our recent cover story.