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Find Clinical Drug Pipeline Developments & Deals by Verve Therapeutics
VERVE-101 is a PCSK9 gene editing program trial was halted, due to safety concerns occured in patients with heterozygous familial hypercholesterolemia.
The net proceeds will fund the continued clinical development of VERVE-101 and preclinical and clinical development of VERVE-102 and VERVE-201, to conduct research and development activities.
VERVE-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood LDL-C. It is being evaluated for the treatment of heterozygous familial hypercholesterolemia.
Under the terms of the collaboration, Verve will advance the research and development of its preclinical stage in vivo gene editing program targeting lipoprotein(a), an LDL-like particle with apolipoprotein B covalently linked to apolipoprotein(a).
VERVE-101 is a novel, investigational gene editing medicine that permanently turns off the PCSK9 gene in the liver to reduce disease-driving LDL-C. It is being developed initially as a treatment for patients with heterozygous familial hypercholesterolemia (HeFH)
The safety profile observed in the first dose cohort with VERVE-101 is encouraging, and in-line with safety data generated with VERVE-101 in the preclinical studies.
VERVE-201 is designed to permanently turn off the ANGPTL3 gene in the liver, a key regulator of cholesterol and triglyceride metabolism, with a precise A-to-G base pair DNA change.
Under the terms of the collaboration, Verve will advance the discovery, research and certain preclinical development of a novel in vivo gene editing program for the target of interest, with all program costs funded by Vertex.
VERVE-101 utilizes LNP-mediated delivery to target the liver and base editing technology to make a single A-to-G base change at a specific site in the PCSK9 gene in order to disrupt PCSK9 protein production, which subsequently lowers LDL-C levels in the blood.
Verve’s lead product candidate, VERVE-101, is designed to turn off the PCSK9 gene in the liver in order to disrupt blood PCSK9 protein production and thereby reduce blood LDL-C levels, with the goal of reducing a patient’s risk for cardiovascular disease.