Boston-based Verve Therapeutics presented strong findings Nov. 12 for a gene therapy candidate aimed at disabling the gene linked to low-density lipoprotein cholesterol.
The drug candidate is designed as a one-time treatment to "inactivate the PCSK9 gene in the liver to durably lower blood LDL-C," Verve said in a news release. The phase 1 trial recruited 10 patients with familial hypercholesterolemia, a genetic abnormality. Participants were either given sub-therapeutic doses, such as 0.1 or 0.3 milligrams per kilogram, or potentially therapeutic doses, including 0.45 or 0.6 milligrams per kilogram.
Among the patients treated with possible therapeutic doses, LDL cholesterol was reduced between 39% and 55%. Eli Lilly plans to help with future studies of the experimental treatment after the drugmaker invested $310 million to sponsor the research and acquire rights, according to The New York Times.