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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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