Source
StreetInsider
- Presented improved editing data in non-human primates (NHP) for sickle cell disease (SCD), demonstrating a single dose of Gene Writer achieved an average of 85% of long-term hematopoietic stem cells (LT-HSCs) with at least one allele edited on single cell analysis, exceeding the anticipated curative threshold
- Reported first demonstration of permanent chimeric antigen receptor (CAR) integration in NHP with a single dose of Gene Writer delivered as all-RNA in T cell-directed lipid nanoparticles (LNP), which expanded to ~60% of circulating T cells and resulted in meaningful B cell clearance in blood and lymph nodes
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