Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June

Rhea-AI Summary

Editas Medicine (NASDAQ: EDIT) reported promising preclinical data for its in vivo gene editing treatment for sickle cell disease and beta thalassemia. Using proprietary targeted lipid nanoparticle (tLNP) delivery, the company achieved 58% mean editing in hematopoietic stem cells (HSCs) at five months after a single dose in non-human primates, significantly exceeding the 25% threshold needed for therapeutic benefit. The treatment targets HBG1/2 promoters to increase fetal hemoglobin production, mimicking natural mechanisms. Notably, the biodistribution data showed significant liver de-targeting compared to standard LNPs. The data will be presented at the European Hematology Association 2025 Congress in Milan, Italy.

Positive

• Achieved 58% mean editing levels, well above the 25% threshold required for therapeutic benefit
• Single intravenous dose administration shows long-lasting effects at 5 months
• Demonstrated significant liver de-targeting compared to standard LNPs, improving safety profile
• Treatment approach already clinically validated through previous trials with reni-cel

Negative

• Still in pre-clinical development phase
• Results limited to non-human primate studies
• Potential competition from existing sickle cell disease treatments

Insights

Biotechnology Researcher

Editas Medicine's NHP study demonstrates remarkable in vivo editing efficiency that could revolutionize sickle cell disease treatment approaches.

Editas Medicine's latest data represents a significant breakthrough in gene editing for hemoglobinopathies. The company achieved 58% mean editing in hematopoietic stem cells (HSCs) at the HBG1/2 promoter region in non-human primates - more than double the 25% threshold considered necessary for therapeutic benefit. This is particularly impressive as it was achieved with a single intravenous dose and maintained for five months, suggesting durability of effect.

The technical achievement here is substantial. Their proprietary targeted lipid nanoparticle (tLNP) delivery system successfully targets HSCs while showing reduced liver accumulation compared to standard LNPs. This biodistribution profile addresses a key challenge in gene therapy delivery systems. The editing strategy itself is sound - mimicking natural hereditary persistence of fetal hemoglobin (HPFH) by targeting the HBG1/2 promoters with AsCas12a to upregulate fetal hemoglobin.

What makes this approach potentially transformative is the shift from ex vivo to in vivo editing. Current approved gene therapies for sickle cell disease require harvesting a patient's cells, modifying them in a lab, and reinfusing them - a complex, expensive process requiring specialized centers. An in vivo approach that directly edits cells within the patient's body could dramatically simplify treatment, reduce costs, and expand access globally, particularly in regions with high sickle cell disease prevalence but limited healthcare infrastructure.

While these results are in non-human primates and substantial work remains before human trials, the data strongly supports continued development. The 58% editing efficiency substantially exceeds therapeutic thresholds, and the mechanism builds upon their clinically validated reni-cel program, which has already demonstrated hemoglobin increases in human trials using the same editing target.

Achieved 58% mean editing at five months after a single dose using high efficiency HSC delivery, demonstrating therapeutically relevant editing levels using a clinically validated strategy.

Achievement supports development of a novel, in vivo approach to treating sickle cell disease and beta thalassemia.

CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today shared new in vivo data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) with a single dose of proprietary targeted lipid nanoparticle (tLNP) in non-human primates (NHPs). This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for the treatment of sickle cell disease and beta thalassemia. The Company reported these data in a presentation available today and will detail the data in a poster session on Saturday, June 14^th 6:30 - 7:30 p.m. CEST (12:30 – 1:30 p.m. EDT) at the European Hematology Association (EHA) 2025 Congress in Milan, Italy.

In this study, the Company’s proprietary tLNP formulation delivered HBG1/2 promoter editing cargo to HSCs in NHPs. Latest data from this ongoing NHP study showed that at five months a single intravenous administration of Editas’ tLNP resulted in mean on-target editing levels in the HBG1/2 promoter region of 58% in HSCs: well exceeding the predicted editing threshold of ≥25% required for therapeutic benefit. In addition to achieving therapeutically relevant editing levels, the biodistribution data in NHPs with Editas’ tLNP continue to show significant de-targeting of the liver in contrast to standard LNPs.

“These data from our in vivo HSC program confirm our ability to achieve high efficiency delivery, therapeutically relevant editing levels and favorable biodistribution in NHPs. These data validate the further development of Editas’ proprietary HSC-tLNP for editing of the HBG1/2 promoters for the treatment of sickle cell disease and beta thalassemia,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine.

Editas Medicine’s in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to edit with high efficiency and minimize off-target editing. Editing the HBG1/2 promoters with AsCas12a with the investigational medicine reni-cel led to robust increases in HbF and total hemoglobin (Hb) in clinical trials.

The presentation details are listed below. Abstracts can be accessed on the EHA website, and the presentation will be posted on the Editas Medicine website during the conference.

Poster Presentation Details:
Title: Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for β-hemoglobinopathies
Date/Time: Saturday, June 14, 2025, 6:30 - 7:30 p.m. CEST/ 12:30 – 1:30 p.m. EDT
Location: Allianz MiCo, Milano Convention Centre
Session: Poster Session 2

About Editas Medicine
As a pioneering gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, 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.

Media and Investor Contacts:
media@editasmed.com
ir@editasmed.com

FAQ

What editing efficiency did Editas Medicine achieve in their EDIT stock HBG1/2 promoter study?

Editas Medicine achieved 58% mean editing levels in HSCs at five months after a single dose, exceeding the 25% threshold required for therapeutic benefit.

How does Editas Medicine's new treatment target sickle cell disease?

The treatment uses targeted lipid nanoparticles to deliver gene editing cargo that modifies HBG1/2 promoters, mimicking natural mechanisms to increase fetal hemoglobin production.

What makes Editas Medicine's tLNP delivery system unique for EDIT stock?

The proprietary tLNP system shows significant de-targeting of the liver compared to standard LNPs, potentially improving safety, and achieves high-efficiency delivery to hematopoietic stem cells.

When will Editas Medicine present their HBG1/2 promoter editing data?

The data will be presented at the European Hematology Association 2025 Congress in Milan, Italy, on June 14th, 2025, from 6:30-7:30 p.m. CEST.

What diseases could benefit from Editas Medicine's new gene editing treatment?

The treatment is being developed for both sickle cell disease and beta thalassemia patients.