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Editas Medicine Reports New Preclinical Data Demonstrating Progress of EDIT-401 as Potential Treatment for Hyperlipidemia at the American Society of Gene and Cell Therapy 2026 Annual Meeting
New data demonstrate promising preclinical safety profile and durable LDL-C lowering of ≥90% with single dose of EDIT-401 in non-human primates through ~6
About this update from Editas Medicine, Inc.
[{"type":"text","content":"New data demonstrate promising preclinical safety profile and durable LDL-C lowering of ≥90% with single dose of EDIT-401 in non-human primates through ~6 months\nCAMBRIDGE, Mass., May 14, 2026 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company developing transformative medicines for serious diseases, shared new preclinical data supporting the continued advancement of Editas’ lead in vivo development candidate, EDIT-401, and its potential as a one-time treatment for hyperlipidemia, as well as the broader potential of the Company’s differentiated upregulation strategy. The data is being presented this week at the 2026 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Boston, including one oral presentation and two poster presentations, as well as one oral presentation at TIDES USA 2026: Oligonucleotide and Peptide Therapeutics Conference. Key EDIT-401 data presented include: In an oral presentation at ASGCT, Editas reported that a single dose of EDIT-401 achieved ≥90 percent mean LDL-C reduction across all dose groups in non-human primates (NHPs). ≥90 percent mean LDL-C reduction was achieved with only moderate levels (10-40 percent) of functional editing of LDLR alleles and ≥6-fold mean increase in hepatic LDLR protein.LDL-C lowering was rapid and remained durable across evaluated dose levels (1.5 mg/kg-3.0 mg/kg) through ~6 months.Promising preclinical safety profile with no adverse clinical observations at therapeutically relevant dose (1.5 mg/kg).The highest delivery of EDIT-401 was observed in the hepatocytes as compared to other non-target tissues with undetectable oocyte delivery. In an oral presentation at TIDES, Editas presented data demonstrating EDIT-401 dose-dependent LDL-C reduction in NHPs.In a poster presentation at ASGCT, Editas reported that data evaluating pharmacokinetics and pharmacodynamics of a single dose of EDIT-401(mu) across multiple dose levels in heterozygous Ldlr loss-of-function mice and wildtype mice support that dose adjustments may not be needed to achieve LDL-C lowering in Heterozygous Familial Hypercholesterolemia (HeFH) patients. Additional in vivo upregulation findings from a poster presentation at ASGCT include: Data support leveraging DNA large language prediction models (DNA-LLM) to accelerate and streamline the pursuit of ge...