Louise

Louise asked about the go/no-go decision for BEAM-103 in healthy volunteers and how its editing efficiencies compare to the in vivo editing in HSCs.

Answer

CEO John Evans and President Giuseppe Ciaramella responded. Giuseppe Ciaramella clarified that the BEAM-103 healthy volunteer study doses only the anti-CD117 antibody component of the ESCAPE technology to confirm safety and develop a PK/PD model, without any editing. He confirmed that when combined with the additional edit, the editing efficiency is very high and comparable to other approaches.

Louise inquired about the go/no-go decision criteria for BEAM-103 in healthy volunteers and how its editing efficiencies compare to the in vivo HSC editing program.

Answer

CEO John Evans and President Giuseppe Pino Ciaramella clarified that the BEAM-103 healthy volunteer study involves dosing only the anti-CD117 antibody component of the ESCAPE technology to confirm safety and develop a PK/PD model for dosing in sickle cell patients, with no editing occurring in this study. They confirmed that the editing efficiency in combination with the additional edit (protecting edited cells from antibody binding) is very high and comparable to in vivo delivered technology.