- Verve Therapeutics Inc (NASDAQ:VERV) has reported new preclinical data from 36 non-human primates (NHPs) with its lead candidate, VERVE-101.
- VERVE-101 is a potential single-course gene-editing treatment for atherosclerotic cardiovascular disease (ASCVD).
- The data will be presented today at the TIDES USA Oligonucleotide & Peptide Therapeutics Conference.
- VERVE-101 is being developed initially for heterozygous familial hypercholesterolemia (HeFH), a potentially fatal genetic heart disease.
- VERVE-101 is designed to inactivate the PCSK9 gene, resulting in a sustained reduction in PCSK9 protein levels and increased LDLR expression, leading to lower LDL-C levels and reduced risk for ASCVD.
- 70% mean editing at the PCSK9 target site in NHPs treated with 1.5 mg/kg VERVE-101 (n=22) as observed in liver biopsies at two weeks.
- Significant, durable reductions in PCSK9 and LDL-C levels were seen after a single dose at two different dose levels when measured at day 14 and day 180.
- Sustained editing of PCSK9 in regenerated liver lobes at 95 days post-treatment was observed.
- No significant off-target editing was observed in human liver cells.
- A generally well-tolerated profile with VERVE-101 at all doses was seen, but mild transient elevated liver enzymes and no impact on glucose levels were observed.
- The Company plans to submit IND for human trials in 2022.
- Price Action: VERV stock is up 0.81% at $47.40 during the market session on the last check Thursday.
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