Verve Therapeutics Posts Updated Preclinical Data For Gene-Editing Therapy In Cardiovascular Disease
Verve Therapeutics Inc (NASDAQ: VERV) announced new preclinical data supporting the nomination of the company's second product candidate, VERVE-201.
VERVE-201 is designed to permanently turn off the ANGPTL3 gene in the liver, a key regulator of cholesterol and triglyceride metabolism.
Verve is initially developing VERVE-201 for homozygous familial hypercholesterolemia (HoFH), a rare genetic subtype of atherosclerotic cardiovascular disease (ASCVD) characterized by extremely high blood low-density lipoprotein cholesterol (LDL-C).
Also Read: Verve Therapeutics, Vertex Join Forces For Liver Disease-Focused Gene Program.
The preclinical studies showed potent, on-target editing of the ANGPTL3 gene with no detectable off-target and no detectable structural variants as assessed using high-coverage whole genome optical mapping.
Verve also conducted a non-human primate (NHP) study (n=9) demonstrating potent liver ANGPTL3 editing of 54-57% and reductions in blood ANGPTL3 protein of 95-98% across three dose levels.
Additionally, Verve shared an update on previously reported data with an ANGPTL3 precursor formulation targeting the same base editing outcome in NHPs.
These data demonstrated durable editing in the liver of treated NHPs (n=4) of 61% over two years following dosing, which was identical to editing levels measured via a liver biopsy on day 15.
Price Action: VERV shares are up 3.17% at $34.54 on the last check Monday.
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