Editas Medicine, Inc. (NASDAQ: EDIT) announced on October 10, 2025 that it presented new in‑vivo proof‑of‑concept data for its experimental LDL‑C lowering therapy, EDIT‑401, at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress in Seville, Spain, held on October 9, 2025.
The preclinical data, derived from non‑human primate studies, showed a ≥90% mean reduction in low‑density lipoprotein cholesterol (LDL‑C) following a single intravenous dose of EDIT‑401. The therapy employs Editas’s proprietary AsCas12a enzyme and targeted lipid nanoparticle delivery to upregulate the LDL receptor (LDLR), a novel approach that could provide durable, one‑time treatment for hyperlipidemia.
This milestone is significant for Editas because it demonstrates a measurable therapeutic effect in a relevant animal model, validates the company’s upregulation strategy, and positions EDIT‑401 as a potential best‑in‑class candidate for LDL‑C reduction. The data support the company’s broader pipeline strategy and may influence future clinical development plans and investor expectations.
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