Recent genetic discoveries and increasing sophistication of gene editing technologies are enabling a potential transformation of the treatment paradigm for atherosclerotic cardiovascular disease (ASCVD).
Building on the Foundation of Genetic Discoveries
In the last decade, our expanding understanding of human genetics led to an important finding – existence of naturally occurring gene variants that have been shown to dramatically lower some individuals' lifetime risk of ASCVD and protect against heart attack. This finding informed our scientific approach, which aims to mimic these natural disease resistance mutations by inactivating specific genes to lower blood lipids through a single-course therapy, thereby reducing the risk of ASCVD.
Matching Gene Editing Technology to the Target
Gene editing technologies have also rapidly advanced in the last decade. Once a target has been selected based on genetic and pharmacologic data, we are technology flexible and compare multiple editing technologies to evaluate what’s best for each target. This was our approach for our first two programs and what we plan to do for future programs.
Our initial programs, VERVE-101, VERVE-102, and VERVE-201, use a form of gene editing known as base editing. Base editing is a gene editing approach that enables precise and efficient editing at the single-base level in the genome without making a double-stranded break in the DNA.