Editas Medicine reports 90% LDL-C reduction in preclinical studies

CAMBRIDGE, Mass. - Editas Medicine, Inc. (NASDAQ:EDIT), whose stock has surged over 260% in the past six months according to InvestingPro data, presented preclinical data for its experimental gene therapy EDIT-401 showing significant reductions in LDL cholesterol levels at the European Society of Gene and Cell Therapy Congress in Seville on Thursday.

The data demonstrated that a single dose of EDIT-401 achieved at least 90% reduction in LDL cholesterol within 48 hours in non-human primates. Similar reductions were observed in mouse models with high baseline LDL-C and reduced LDL receptor function. The company, which maintains a strong balance sheet with more cash than debt according to InvestingPro analysis, is well-positioned to advance its research programs.

The therapy uses CRISPR/Cas9 gene editing technology delivered via lipid nanoparticles to target regulatory elements in the LDL receptor gene. According to the company, this approach resulted in a six-fold increase in LDL receptor protein in non-human primate livers.

The treatment’s cholesterol-lowering effect was maintained throughout a three-month study in mouse models, suggesting potential durability.

"The in vivo proof-of-concept data presented today reinforce the potential impact of our differentiated upregulation strategy," said Linda C. Burkly, Executive Vice President and Chief Scientific Officer at Editas Medicine, in a press release statement.

EDIT-401 is being developed as a potential one-time therapy for patients with high LDL cholesterol, though the company did not provide a timeline for human clinical trials.

The gene therapy aims to upregulate the LDL receptor by increasing mRNA stability through the disruption of negative regulatory elements.

The data was presented as part of an oral presentation at the 32nd Annual European Society of Gene and Cell Therapy Congress. With the company’s next earnings report scheduled for November 10th and multiple additional insights available through InvestingPro’s comprehensive research reports, investors can access deeper analysis of Editas Medicine’s financial health and growth prospects.

In other recent news, Editas Medicine has been actively engaged in several noteworthy developments. The company is set to present its research on LDL-cholesterol lowering using CRISPR gene editing at the European Society of Gene and Cell Therapy Congress. This research involves the upregulation of LDLR in mice and non-human primates, showcasing promising advancements in gene editing techniques. In parallel, Editas Medicine’s EDIT-401 program has drawn significant attention, with H.C. Wainwright maintaining a Buy rating and setting a price target of $5.00, citing the program’s potential for substantial LDL-C reduction.

Additionally, Wells Fargo has raised its price target for Editas Medicine to $4.00, maintaining an Equal Weight rating based on encouraging preclinical data for EDIT-401. This data demonstrated a 90% reduction in LDL-C levels in non-human primates after a single dose, with no severe adverse effects noted. Furthermore, Baird has increased its price target to $6.00, maintaining an Outperform rating due to the company’s progress in its pipeline, which aligns with expectations. These developments highlight Editas Medicine’s ongoing efforts in advancing its gene editing capabilities and potential therapeutic applications.

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