VERV - Verve Therapeutic Provides First Look At Gene-Editing Drug For Inherited High 'Bad' Cholesterol Shares Fall | Benzinga
Verve Therapeutics Inc (NASDAQ: VERV) released the first human proof-of-concept data for in vivo base editing from the ongoing heart-1 phase 1b trial of VERVE-101 from the first ten people treated.
The data were presented at the American Heart Association's annual meeting.
All ten were enrolled with heterozygous familial hypercholesterolemia (HeFH), an inherited condition that causes severe elevations in low-density lipoprotein (LDL) cholesterol.
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.
Among three participants given the two highest tested doses, treatment with Verve's therapy lowered LDL ...