Editas Medicine eyes first human trial for CRISPR cholesterol therapy EDIT-401. Editas Medicine (NASDAQ:EDIT) is preparing to begin a first-in-human study of EDIT-401, an in vivo CRISPR-edited medicine designed to lower LDL cholesterol in patients with heterozygous familial hypercholesterolemia, Chief Executive Officer Gilmore O'Neill said during a Wells Fargo discussion. O'Neill said the company is focused entirely on in vivo CRISPR-edited medicines and is prioritizing programs that could produce differentiated efficacy, use capabilities unique to gene editing and offer measurable biomarkers for early clinical proof of concept. The company is also emphasizing manufacturing costs and accessibility, he said, noting that Editas no longer develops cell-based therapies. EDIT-401 targets LDL receptor expression. Discover more Defense contractor directory Military technology insights Defense industry analysis EDIT-401 is intended to increase production of the LDL receptor in liver cells, allowing the receptor to remove more LDL cholesterol from the bloodstream. The treatment is designed as a single intravenous infusion. According to O'Neill, EDIT-401 reduced LDL cholesterol, lipoprotein(a), or Lp(a), and apolipoprotein B by a mean of 90% in non-human primates. The company said it has observed at least a sixfold increase in LDL receptor expression in the livers of non-human primates. The program was informed by naturally occurring gain-of-function variants observed in Icelandic and French families. Those variants involve deletions in the three-prime untranslated region of the LDLR gene, which can stabilize messenger RNA and support increased LDL receptor protein production. Editas selected guide RNAs intended to create a comparable, but not identical, deletion. O'Neill said the company's preclinical findings suggest that editing roughly 15% of alleles could produce substantial cholesterol lowering in non-human primates. Company modeling indicates that most of the editing at that level is monoallelic rather than biallelic, meaning the company may not need to edit the entire hepatocyte population to achieve the desired effect. Clinical study planned in Australia. Editas has submitted documents to human research ethics committees in Australia and is in discussions with those committees, O'Neill said. The company remains on track to dose the first patients this year. The Phase 1 trial is expected to enroll patients with heterozygous familial hypercholesterolemia who remain above LDL cholesterol targets despite intensive standard-of-care treatment. The study will have two parts: * Part 1 will use serial dose-escalation cohorts under a 3+3 design, with at least three participants per cohort and the option to enroll three additional patients if warranted. * The company anticipates at least four cohorts in Part 1. * Part 2 will expand the selected dose into a broader patient population. O'Neill said Editas expects to share safety data in the first quarter of 2027 and aims to report top-line data from Part 1 later in 2027. The company expects to enter the U.S. in 2027, following what O'Neill described as constructive pre-IND interactions with the Food and Drug Administration. He said the main limiting factor for U.S. entry is preparing manufacturing documentation. The company expects a roughly three-to-one dose translation from non-human primates to humans, based on available experience across in vivo editing programs. O'Neill said a 1.5 mg/kg non-human primate dose could translate to an approximate 0.5 mg/kg to 0.6 mg/kg human dose, though clinical results will determine the ultimately effective dose. Safety and Lp(a) considerations. Editas is working with Genevant on a lipid nanoparticle, or LNP, delivery system for EDIT-401. O'Neill said the LNP is unique to the program, though most of its components have previously been used in humans. In preclinical toxicology studies, the company observed minimal liver-enzyme increases at therapeutically relevant doses, with transaminase changes comparable with saline control animals. At higher doses, liver-enzyme increases resolved within days and returned to normal range within a week or less, he said. O'Neill said recent results from Novartis' HORIZON study do not alter Editas' strategy. He characterized the Lp(a) reduction associated with EDIT-401 as an additional potential benefit, while emphasizing that the company's primary objective is substantial LDL cholesterol lowering. He also said genetic and clinical evidence supports lowering LDL cholesterol to very low levels in high-risk patients. Cash runway and pipeline. Chief Financial Officer Amy Parison said Editas ended the third quarter with $212 million in cash and expects its cash runway to extend into the second half of 2028. She said the company plans to direct capital toward advancing EDIT-401 through both parts of its Phase 1 study and establishing proof of concept in humans. Beyond EDIT-401, O'Neill said Editas has an in vivo hematopoietic stem cell program in discovery and other early-stage gain-of-function editing programs. The company chose to prioritize EDIT-401 last summer, he said, while continuing to optimize its earlier programs. O'Neill added that future liver-targeted programs could potentially leverage the LNP, messenger RNA and manufacturing work developed for EDIT-401. About Editas Medicine (NASDAQ:EDIT). Editas Medicine is a clinical-stage biotechnology company focused on translating the power of gene editing into a new class of transformative genomic medicines. Founded in 2013 and headquartered in Cambridge, Massachusetts, the company leverages proprietary CRISPR/Cas9 and CRISPR/Cas12a (Cpf1) platforms to develop therapies aimed at correcting disease-causing genetic mutations. Editas Medicine's research and development efforts span multiple therapeutic areas, including inherited retinal diseases, hemoglobinopathies, and oncology. The company's pipeline includes EDIT-101, a lead candidate designed to treat Leber congenital amaurosis type 10 (LCA10), which has entered early-stage clinical trials, and EDIT-301, targeting sickle cell disease and β-thalassemia using an ex vivo editing approach.

Editas Medicine has appointed Dan Ory as chief medical officer, effective immediately. Ory brings over 25 years of experience in cardiovascular and genetic medicine to oversee clinical development strategy as the company advances EDIT-401, its lead in vivo candidate for treating hyperlipidemia, towards clinical trials. Before joining Editas, Ory served as chief medical officer at Arbor Biotechnologies, where he led the global clinical trial for the company's lead in vivo gene editing programme. He previously held the same role at Casma Therapeutics, focusing on rare genetic and neurodegenerative diseases. Earlier in his career, Ory spent over two decades at Washington University School of Medicine, where he researched cholesterol metabolism and Niemann-Pick disease type C.

Editas Medicine appoints Dan Ory, M.D., as Chief Medical Officer. Experienced biotechnology executive with more than 25 years of leadership in cardiovascular and genetic medicine to lead clinical development Appointment strengthens executive leadership team as Editas advances EDIT-401 toward clinical development CAMBRIDGE, Mass., Sept. 08, 2026 (GLOBE NEWSWIRE) - Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company focused on developing transformative medicines for serious diseases, today announced the appointment of Dan Ory, M.D., as Chief Medical Officer, effective today. With more than 25 years of experience spanning biotechnology leadership, cardiovascular medicine, and the development of genetic medicines, Dr. Ory will oversee the company's clinical development strategy and operations as Editas advances EDIT-401, its lead in vivo development candidate for the potential treatment of hyperlipidemia, toward the clinic, while supporting the progression of the company's broader pipeline. Prior to joining Editas, Dr. Ory served as Chief Medical Officer at Arbor Biotechnologies, where he oversaw the global clinical trial for the company's lead in vivo gene editing program. Previously, he served as Chief Medical Officer at Casma Therapeutics, leading clinical development programs focused on rare genetic and neurodegenerative diseases. Before joining industry, Dr. Ory spent more than two decades at Washington University School of Medicine in St. Louis, where he served as the Alan A. and Edith L. Wolff Professor of Cardiology and conducted research in cholesterol metabolism and Niemann-Pick disease type C (NPC), helping advance the understanding of cholesterol homeostasis and its role in cardiovascular disease. "Dan joins Editas at an exciting time as EDIT-401 nears clinical development for the potential treatment of hyperlipidemia, and as we continue advancing our leadership in in vivo gene editing," said Gilmore O'Neill, M.B., M.M.Sc., President and Chief Executive Officer of Editas Medicine. "He brings deep expertise in cardiovascular medicine and the development of genetic medicines, together with a proven track record of advancing innovative therapies from scientific discovery through clinical development. His expertise in cardiovascular disease and cholesterol metabolism, combined with his experience leading clinical development for innovative genetic medicine programs, including in vivo gene editing, will strengthen our ability to execute our clinical strategy and advance our mission of developing transformative in vivo gene editing medicines for patients." "I am excited to join Editas as the company advances EDIT-401 toward the clinic," said Dr. Ory. "The opportunity to apply in vivo gene editing to cardiovascular disease, an area where significant unmet need remains, represents an exciting new frontier in medicine. EDIT-401 has the potential to redefine the treatment paradigm as a best-in-class therapeutic for hyperlipidemia through a one-time gene editing approach, and I have been impressed by the strength of the science and the talented team behind it. I look forward to working alongside my colleagues to advance EDIT-401 into the clinic, progress the broader pipeline, and work to ultimately deliver transformative medicines for patients living with serious diseases." About Dan Ory, M.D. Dr. Ory, M.D., is an accomplished biotechnology executive with more than 25 years of leadership in cardiovascular and genetic medicine. Most recently, Dr. Ory served as Chief Medical Officer at Arbor Biotechnologies, where he led the company's clinical development strategy and advancement of its next-generation gene editing pipeline. Previously, he served as Chief Medical Officer at Casma Therapeutics, where he oversaw clinical development programs focused on rare genetic and neurodegenerative diseases. Before transitioning to industry, Dr. Ory was the Alan A. and Edith L. Wolff Professor of Cardiology at Washington University School of Medicine in St. Louis, where his laboratory made significant contributions to the understanding of cholesterol metabolism and advanced the field of Niemann-Pick disease type C (NPC), a rare neurodegenerative cholesterol storage disorder. He also led multiple clinical trials in NPC disease and was scientific co-founder of Vtesse Therapeutics, a rare disease company. Clinically, Dr. Ory's practice was focused on preventive and diagnostic cardiology, specializing in cardiovascular risk assessment and the application of stress echocardiography and nuclear perfusion imaging to guide the evaluation and management of patients at risk for coronary artery disease. During his academic career, he authored more than 160 peer-reviewed publications and is an inventor on numerous patents related to cardiovascular and rare disease research. He was elected a Fellow of the American Association for the Advancement of Science (AAAS) and is a member of both the American Society for Clinical Investigation (ASCI) and the Association of American Physicians (AAP). Dr. Ory received an A.B. from Harvard College and an M.D. from Harvard Medical School. He completed postdoctoral training at the Whitehead Institute at MIT, an internal medicine residency at Brigham and Women's Hospital, and a fellowship in cardiology at Massachusetts General Hospital. About Editas Medicine As a pioneering gene editing company, Editas Medicine is focused on translating the power and potential of CRISPR genome editing systems into a robust pipeline of transformative in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize durable, precision in vivo gene editing medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute's Cas12a patent estate and Broad Institute and Harvard University's Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com. Forward-Looking Statements This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "target," "should," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation, timing, progress, and results of preclinical studies and clinical trials; uncertainty regarding availability and timing of results from preclinical studies and clinical trials; uncertainties relating to planned regulatory submissions to initiate clinical trials, including that results of preclinical studies will warrant such submissions or that regulatory agencies may require additional preclinical studies, that regulatory submissions shall occur on the expected timelines and that regulatory authorities will provide clearance for trials to be initiated on the expected timelines or at all; and uncertainties as to whether the Company's cash resources are sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements for the period anticipated. 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