VT-20101 is an Open-label, Phase 1b, Single-ascending dose study that will evaluate the safety of VERVE-201 administered to patients with Refractory Hyperlipidemia.
VERVE-201 uses base-editing technology designed to inactivate the expression of the ANGPTL3 gene in the liver and lower circulating low-density lipoprotein cholesterol (LDL-C) and triglycerides. This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 in this patient population.
Who can participate
Inclusion Criteria:
* Refractory hypercholesterolemia
* Refractory hypertriglyceridemia
Exclusion Criteria:
* Active or history of chronic liver disease
* Current treatment with monoclonal antibody targeting ANGPTL3 or prior treatment within specified timeframe
* Clinically significant or abnormal laboratory values as defined by the protocol
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