Preclinical studies achieved therapeutically relevant gene editing levels of the HBG1/2 promoter & favorable biodistribution profile in non-human primates using a clinically validated editing strategy
Data reinforces continued development as a potentially transformative, in vivo approach to treating sickle cell disease and beta thalassemia
CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today announced that new data from a study in non-human primates (NHPs) has been accepted for a poster presentation at the European Hematology Association (EHA) 2025 Congress being held June 12-15, 2025, in Milan, Italy.
Key delivery, editing, and biodistribution data from an ongoing study in NHPs will be shared, including:
- High efficiency HSC delivery of editing cargo.
- Therapeutically relevant editing levels in the HBG1/2 promoter region exceeding the predicted editing threshold of ≥25% required for therapeutic benefit with a single dose.
- Favorable biodistribution with Editas’ tLNP, demonstrating significant de-targeting of the liver in contrast to standard LNPs.
“We look forward to sharing further data from our in vivo HSC program at EHA, demonstrating therapeutically relevant editing levels and favorable biodistribution in NHPs. These data warrant 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, and we are encouraged by our significant progress towards the clinic,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine.
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 Enables In Vivo HBG1/2 Genome Editing In Non-Human Primates
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.
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