“Gene Editing” Verve Therapeutics develops a new generation of gene editing therapy VERVE-101. Early data shows that it can effectively reduce cholesterol levels in HeFH patients and reduce the risk of heart disease and other diseases.
Read in seconds:Verve Therapeutics announced on November 12 thatNext-generation gene editing therapy VERVE-101ofClinical phase Ib trial heart-1Early data onHeterozygous familial hypercholesterolemia (HeFH)The patient hasVERVE-101 treatment significantly reduces cholesterol levels in the body.
According to trial data presented by Verve Therapeutics at the 2023 American Heart Association Scientific Sessions, two people receiving VERVE-101Potential therapeutic dose 0.45 mg/kgof patients, whoseLow-density lipoprotein cholesterol (LDL-C) dropped by an average of 39% and 48%while one acceptsMaximum dose 0.6 mg/kgof patients, whoseLDL-C was reduced by 55%displayA single infusion of VERVE-101 produces a dose-dependent decrease in LDL-C.
htKIt is a genetic disease mainly caused by pathogenic mutations in related genes including PCSK9.PCSK9 protein affects blood cholesterol concentrationswhich in turn causes HeFH patients toDangerously high cholesterol levels can lead to stroke, heart disease or heart attack.
VERVE-101throughChemically modify a single base in the PCSK9 gene and turn the gene offTo reduce overall LDL-C levels, Verve also confirmed in the heart-1 trial that VERVE-101 can significantly reduce PCSK9 protein expression in the blood of two patients who received a dose of 0.45 mg/kg.PCSK9 protein levels were reduced by 59% and 84%whereas patients receiving a maximum dose of 0.6 mg/kgPCSK9 protein levels also dropped by as much as 47%.
Verve Therapeutics stated that its gene-editing drug candidates will not double-strand DNA in order to change bases.Avoids many of the side effects and off-target risks associated with CRISPR/Cas9 systems.The company will now enroll additional patients into potentially therapeutic dose groups in the heart-1 trial, which is expected toSelect a single dose and initiate and complete an expansion cohort trial at that dose in 2024.
Source: BioSpace, Fierce Biotech, Verve Therapeutics
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Tags: Gene Editing Verve Therapeutics develops generation gene editing therapy VERVE101 Early data shows effectively reduce cholesterol levels HeFH patients reduce risk heart disease risks stop biotechnology investment Genet Viewpoint