Verve Therapeutics' Gene-Editing Therapy Shows Encouraging Preclinical Action In Cardiovascular Disease

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  • Verve Therapeutics Inc 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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