SAIL-0804

SAIL-0804 is an investigational in vivo CAR-T therapy developed by Sail Biomedicines for the treatment of autoimmune diseases. It utilizes proprietary Endless RNA (eRNA), a circular RNA technology optimized for enhanced stability and expression, delivered via targeted lipid nanoparticles (tLNPs) engineered for selective T cell delivery. Upon intravenous administration, SAIL-0804 transiently reprograms endogenous T cells to express an anti-CD19 chimeric antigen receptor (CAR), enabling deep and broad B cell depletion—including pro-B and pre-B progenitor cells—without the need for lymphodepletion or ex vivo cell harvesting. The therapy achieves efficient transfection of both CD4+ and CD8+ T cells, resulting in potent cytotoxic activity against B cells while avoiding genomic integration. Preclinical studies have demonstrated rapid B cell ablation with subsequent repopulation by mostly immature phenotypes, suggesting potential for immune reset and long-term remission without sustained immunosuppression[1][2][6][7].

Other names

Sail Biomedicines in vivo CAR-TeRNA in vivo CAR-T