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Phase 1/2 clinical trial of EDIT-401 cleared to initiate dosing in patients who require additional LDL-C lowering 

Company remains on track to begin patient dosing this year and report initial safety and tolerability data in the first quarter of 2027

New EDIT-401 preclinical data demonstrating durability of LDL-C reduction to be presented in oral presentation at AHA Scientific Sessions 2026

CAMBRIDGE, Mass., Oct. 08, 2026 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company focused on developing transformative medicines for serious diseases, today announced it has received approval to initiate the Phase 1/2 Strive™ clinical trial of EDIT-401 in patients with Heterozygous Familial Hypercholesterolemia (HeFH) in Australia. The trial has received Human Research Ethics Committee (HREC) approval and completed the Clinical Trial Notification (CTN) process with Australia’s Therapeutic Goods Administration (TGA) and has also been submitted for review in New Zealand. If approved in New Zealand, patients in both countries will be eligible to enroll in the Strive trial. The Company also announced that new preclinical data, demonstrating durability of LDL-C reduction with EDIT-401, will be released via an oral presentation at the American Heart Association (AHA) Scientific Sessions 2026, taking place November 6-9, 2026, in Chicago, Illinois.

EDIT-401 is an experimental, potentially best-in-class in vivo gene editing medicine for the treatment of hyperlipidemia. Utilizing Editas’ differentiated upregulation approach, EDIT-401 is designed to directly edit the LDLR gene to increase LDLR protein expression and reduce LDL-cholesterol (LDL-C) levels. In preclinical studies, EDIT-401 demonstrated ~90 percent or greater mean reduction in multiple atherogenic lipoproteins, including LDL-C, Lp(a), and ApoB in non-human primates. New preclinical data to be presented at AHA also demonstrated an LDL-C reduction of ~90 percent was maintained over 10 months in an ongoing study in non-human primates.

“The receipt of regulatory and ethics approval for clinical trial initiation in Australia is a significant step in advancing the clinical development of EDIT-401, a potentially transformative in vivo gene editing medicine designed to upregulate LDLR expression and reduce LDL-C levels in patients living with hyperlipidemia. We look forward to initiating the Strive study and generating our first-in-human data as we advance EDIT-401 through key clinical milestones in 2027,” said Dan Ory, M.D., Chief Medical Officer, Editas Medicine. “We are also encouraged by the new EDIT-401 preclinical data to be presented at AHA, which further support its potential to provide durable, lifelong benefits to patients.”

The Strive trial is designed to evaluate the safety, tolerability, and efficacy of a single dose of EDIT-401. The trial is designed in two parts: Part 1 is a single ascending dose, dose-finding, open-label trial. Part 2 will be a single-dose randomized, placebo-controlled expansion study. Editas has selected five clinical trial sites across Australia and New Zealand.

Editas expects to report initial safety and tolerability data in the first quarter of 2027. The Company plans to complete enrollment in Part 1, the dose-finding portion of the Phase 1/2 trial of EDIT-401, with topline safety and efficacy data results available later in 2027.

American Heart Association (AHA) Scientific Sessions 2026 Oral Presentation
Editas will present new preclinical data demonstrating the durability of EDIT-401, supporting its potential as a transformative therapy for people living with hyperlipidemia, at the AHA Scientific Sessions on Sunday, November 8.

Presentation Details:
Title: EDIT-401: In Vivo Gene Editing to Enhance LDLR Function for Durable LDL-C Reduction
Session Date and Time: Sunday, November 8, 2026, 3:30 - 4:45 p.m. CT
Session Title: Translational Gene and Epigenome Therapies For Lipid Management: A New Frontier
Presenter: Dan Ory, M.D., Chief Medical Officer, Editas Medicine

The final presentation can be accessed on the AHA website. A copy of the presentation will also be posted to the “Posters & Presentations” section of the Company’s website during the conference.

About EDIT-401
EDIT-401 is an experimental, potentially best-in-class in vivo gene editing medicine, based on Editas’ differentiated upregulation approach. EDIT-401 is designed to treat hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels. This targeted approach has demonstrated a favorable preclinical profile in both efficacy data and tolerability and supports the potential of EDIT-401 to deliver meaningful clinical outcomes for patients underserved by current lipid-lowering therapies.

About Heterozygous Familial Hypercholesterolemia (HeFH)
Heterozygous Familial Hypercholesterolemia (HeFH) is an inherited genetic disorder that leads to significantly elevated LDL-C levels from an early age. Individuals with HeFH are at high risk of heart disease, heart attack, or stroke if the condition is not identified and treated early. An estimated 1.2 million people in the United States are living with HeFH, though many remain undiagnosed. Elevated LDL-C, also known as hyperlipidemia, is a highly prevalent disease affecting over 70 million patients in the United States alone. Substantial unmet need exists across multiple at-risk segments of patients with hyperlipidemia, including the HeFH population.

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. Forward-looking statements in this press release include statements regarding the initiation, timing, progress and results of the Company’s planned clinical trials, including the Company’s expectation to begin patient dosing in 2026 and complete enrollment in Part 1, the dose-finding portion of the Phase 1/2 trial of EDIT-401, with topline safety and efficacy data results available later in 2027; the timing for the Company’s receipt and presentation of data from its preclinical and planned clinical studies, including reporting initial safety and tolerability data in the clinical trial of EDIT-401 in the first quarter of 2027; and the potential of, and expectations for, EDIT-401. 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. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent the Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.

Investor and Media Contacts: 

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2 gene-editing stocks reshaping hereditary disease treatments 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.

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EDIT-401 Targets LDL Receptor Expression Are Gene Therapy Stocks The Market's Next Big Winners?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.

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