(RTTNews) - Editas Medicine Inc. (EDIT), a gene-editing company, on Tuesday released positive preclinical data for EDIT-401, an in vivo gene-editing system, in treating hyperlipidemia. The data was presented at the 94th European Atherosclerosis Society (EAS) congress.
EDIT-401 is an experimental gene editing system that directly edits the low-density lipoprotein receptor (LDLR) gene to reduce levels of low-density lipoprotein cholesterol (LDL-C). It is being developed as a one-time gene therapy that reduces the lifetime risk of cardiovascular disease.
The preclinical study conducted in non-human primates assessed levels of LDL-C, Lipoprotein a (Lp(a)), a biomarker in atherosclerotic cardiovascular disease (ASCVD), and apoprotein B (ApoB), a key measure of total plaque-causing cholesterol particles and predictive measure for ASCVD.
EDIT-401 was found to affect a =90% mean reduction in LDL-C, and approximately 90% mean reduction in Lp(a) and ApoB levels. Reductions in LDL-C, Lp(a), and Apo(B) were highly correlated, indicating a unified upregulation of the LDLR gene.
A good laboratory practices (GLP) toxicology study is ongoing, with interim results demonstrating that EDIT-401 was well-tolerated, and showed no liver histopathology. Initiation of a clinical study with patients of Heterozygous Familial Hypercholesterolemia (HeFH) is expected later this year.
The company intends to use the data to support an investigational new drug (IND) submission, and reported a successful pre-IND meeting with the U.S. Food and Drug Administration (FDA). A clinical trial notification (CTN) is also planned to the Therapeutic Goods Administration (TGA) in Australia by mid-2026.
Editas announced a proposed underwritten public offering of shares of the company's common stock and pre-funded warrants.
EDIT closed Friday at $2.76, up 3.37%. In the pre-market, shares are trading at $2.45, down 11.23%.
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