Verve’s durable PCSK9 base editor; plus Novartis, PureTech and more

Verve Therapeutics Inc. (NASDAQ:VERV) revealed at the TIDES USA Oligonucleotide & Peptide Therapeutics Conference that its clinical formulation of VERVE-101, a gene editing treatment for patients with heterozygous familial hypercholesterolemia, reduced PCSK9 protein and low-density lipoprotein cholesterol (LDL-C) in non-human primates by 86% and 62%, respectively, 180 days after receiving the higher dose of 1.5 mg/kg. Verve found that the editing persisted in regenerated liver tissue and saw no evidence of off-target editing or editing of germline cells.

The company also provided an update from its non-human primate pilot study using a precursor formulation showing sustained 90% and 60% reductions in blood PCSK9 protein and LDL-C, respectively, at a 15-month follow-up. The company plans to submit an IND and begin clinical trials next year...