Skip to main content
Part of Sickle Cell Disease on Somebody To Talk To

Clinical Trials and Research1 min read

Tessera Therapeutics Showcases New Preclinical Data Advancing In Vivo Program in Sickle Cell Disease and CAR-T Applications at the American Society of Gene and Cell Therapy 29th Annual Meeting

About this article

Published
Further reading
Read More(opens in a new tab)
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
Share