Do Verve's gene edits pass down to patients' children? The FDA wants to know
By Nick Paul Taylor,
Fierce Biotech
| 12. 05. 2022
The FDA has set out its reasons for putting Verve Therapeutics’ high cholesterol gene editing therapy on hold. Officials want additional data to allay concerns that patients could pass on the edited genes to their children before they lift the clinical hold.
Last month, the FDA put Verve’s application on hold and vowed to send an official letter setting out its questions within 30 days. Verve now has the letter—and knows what needs to be done to get the clinical trial of its PCSK9-deactivating drug candidate VERVE-101 off the ground.
The FDA has asked for more preclinical data on potency differences between human and non-human cells, the risks of germline editing and off-target analyses in non-hepatocyte cell types. Officials also want Verve to share data from the heart-1 trial, which continues to enroll patients in New Zealand and the U.K., and to tweak the protocol to mitigate the risks of the therapy.
Specifically, the FDA has asked Verve to incorporate additional contraceptive measures and to increase the length of the staggering interval between dosing of participants. Verve plans to submit...
Related Articles
By Jonathan Matthews, GMWatch | 12.11.2025
In our first article in this series, we investigated the dark PR tactics that have accompanied Colossal Bioscience’s de-extinction disinformation campaign, in which transgenic cloned grey wolves have been showcased to the world as resurrected dire wolves – a...
By Jenny Lange, BioNews | 12.01.2025
A UK toddler with a rare genetic condition was the first person to receive a new gene therapy that appears to halt disease progression.
Oliver, now three years old, has Hunter syndrome, an inherited genetic disorder that leads to physical...
By Simar Bajaj, The New York Times | 11.27.2025
A common cold was enough to kill Cora Oakley.
Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare genetic condition that leaves the body without key white blood cells.
It’s better...
By Rachel Hall, The Guardian | 11.30.2025
Couples are needlessly going through IVF because male infertility is under-researched, with the NHS too often failing to diagnose treatable causes, leading experts have said.
Poor understanding among GPs and a lack of specialists and NHS testing means male infertility...
