Editas Medicine, Inc. · NASDAQ:EDIT
Editas Medicine — ASGCT 28th Annual Meeting 2025
8 slides from Editas Medicine's conference presentation, May 2025
Slide 1: In vivo delivery of HBG1/2 promoter editing cargo to HSCs with lipid nanoparticles
In vivo delivery of HBG1/2 promoter editing cargo to HSCs of humanized mouse and non-human primate with lipid nanoparticles ASGCT 28th Annual Meeting, New Orleans, Louisiana; May 13–17, 2025 Li Li, Peisheng Zhang, Stephen Sherman, Ying Shao, Emily Kaye, Katerina Tsiounis, Jieni Xu, He Wang, Tallie A
Slide 2: Editas' in vivo HSC program uses a clinically validated target and enzyme
Editas’ in vivo HSC program uses a clinically validated target and enzyme Editing HBG1 and HBG2 promoters with AsCas12a leads to HbF induction and correction of anemia Higher % HbF associated with reduction in SCD events3 Targeting Utilizing proprietary HBG1/HBG2 promoters AsCas12a α α α α α α to mi
Slide 3: Editas' in vivo HSC program positioned to be first-in-class and best-in-class
Editas’ in vivo HSC program is positioned to be potentially first-in-class and best-in-class for sickle cell disease and transfusion-dependent beta-thalassemia Gene target with proven clinical efficacy Delivery to HSC cells • Editing the HBG1/2 promoter with our • Targeting moiety to promote uptake
Slide 4: Targeted LNPs achieve efficient delivery of GFP or B2M editing cargo in mice
Administration of targeted LNPs achieves efficient delivery of either GFP or B2M editing cargo in mice engrafted with human CD34+ cells Flow cytometry analyses of GFP Flow cytometry analyses of expression in bone marrow B2M expression in bone marrow 100 100 % of B2M knockout 80 80 % GFP+ 60 60 40 40
Slide 5: Optimization of tLNP platform provides efficient and sustainable HBG1/2 editing in humanized mice
Optimization of tLNP platform provides capability to achieve efficient and sustainable HBG1/2 editing in humanized mouse model HBG1/2 editing in HSPC HBG1/2 editing in HSC LNP composition and 50 LNP composition process development 48% optimization 50 optimization LNP composition and ~36%** 40 proces
Slide 6: Therapeutically relevant HBG1/2 editing levels after single tLNP dose in non-human primates
High efficiency HSC delivery achieved therapeutically relevant HBG1/2 editing levels after a single dose of Editas’ tLNP in non-human primates ~75% GFP+ HSCs observed 24 hours Up to ~47% HBG1/2 editing observed in HSCs at after a single dose of LNP2 3 months after a single dose of LNP2 (moiety 2) 60
Slide 7: Editas' tLNP shows significant de-targeting of the liver in non-human primates
Editas’ tLNP shows significant de-targeting of the liver in non-human primates Standard LNP (comparator) Editas’ tLNP Liver: High delivery in hepatocytes Liver: Minimal in hepatocytes ISH for cargo IHC for protein Editas’ tLNP formulation containing surrogate GFP mRNA cargo was dosed to NHPs as a si
Slide 8: Summary
Summary • Editas’ proprietary LNP formulation with a targeting moiety efficiently delivers either reporter or editing cargo (GFP mRNA or Cas12a mRNA and guide RNA) to HSCs in both a humanized mouse model and NHPs • Administration of a single dose of proprietary tLNP achieved 48% editing of HBG1/2 in