The CD19-directed, non-viral, PD-1 locus-specific, CRISPR-based integrated CAR T cell therapy BRL-201 was associated with a manageable safety profile and elicited early indications of efficacy in patients with relapsed and refractory non-Hodgkin’s lymphoma (NHL), according to results from a first-in-man Phase 1 trial (NCT04213469). No dose-limiting toxicity (DLT) was observed in any of the patient cohorts infused with BRL-201. However, cytopenias were observed at all doses, although most cases resolved without intervention. Furthermore, at the data collection cut-off date of May 17, 2023 and a median follow-up of 29.0 months (range: 21.5 to 36.2), all patients responded to BRL-201 infusion, of whom 85.7% achieved a complete response (CR) and 14.3% a partial response (PR). Of the patients who achieved and maintained a CR, 7 showed continuous responses at the data cut-off. Notably, the 3-month, 6-month and 12-month overall response rates (ORR) were 80.9%, 71.4% and 57.1% respectively.
