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 li

Dravet syndrome has long represented one of the most challenging pediatric epilepsies encountered in neurology and genetic medicine. Caused primarily by loss‑of‑function variants in SCN1A, the disorder emerges in infancy with prolonged febrile seizures and evolves into a lifelong condition marked by treatment‑resistant epilepsy, developmental delay, and significant morbidity. As the gene and cell therapy community gathers for the American Society of Gene and Cell Therapy (ASG