Editas Medicine to Present in vivo HSC Delivery, Editing, and Biodistribution Data at the European Hematology Association 2025 Congress in June
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.,
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
The presentation details are listed below. Abstracts can be accessed on the EHA website, and the presentation will be posted on the
Poster Presentation Details:
Title: Targeted Lipid Nanoparticle Delivery Enables In Vivo HBG1/2 Genome Editing In Non-Human Primates
Date/Time:
Location: Allianz MiCo,
Session: Poster Session 2
About
As a pioneering gene editing company,
Media and Investor Contact: ir@editasmed.com
Source: Editas Medicine, Inc.
