Editas reports in vivo HSC editing up to 48% HBG1/2, exceeding 25% therapeutic threshold

Editas Medicine, Inc.

Single dose of proprietary tLNP achieved up to 47% HBG1/2 editing in NHP HSCs and 48% in humanized mouse long-term HSCs.
Both studies exceeded the ≥25% editing threshold predicted for therapeutic benefit in sickle cell disease and beta thalassemia.
Preliminary biodistribution shows significant liver de-targeting with tLNP vs. standard LNPs.
Data presented at ASGCT 2025; supports development of in vivo gene editing medicine using AsCas12a-based reni-cel approach.
Clinical validation of HBG1/2 promoter editing previously demonstrated with reni-cel leading to increased HbF and total Hb.

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