Verve Therapeutics Posts Updated Preclinical Data For Gene-Editing Therapy In Cardiovascular Disease

Verve Therapeutics Posts Updated Preclinical Data For Gene-Editing Therapy In Cardiovascular Disease
  • Verve Therapeutics Inc 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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