EHA 2023 | Phase I/II study of EDIT-301, a novel gene-edited autologous HSC medicine, in patients with SCD

The EDIT-301 is Phase I/Phase II that is using a very novel and new enzyme called Caspase 12 for gene editing of sickle cell disease. This is an international, multicenter study. We were very happy to give an update today and give preliminary results for the first four patients that received the infusion of the EDIT-301. And it is really exciting to see, even with this very little number of patients, very safe profile that is consistent with a myeloablative regimen that consists from one chemotherapy, busulfan. And the safety profile was consistent with what we would expect, from pancytopenia and some mucositis, and there was no severe grade four toxicity, and there was no side effect related to the infusion of the edited CD34 cells.

And finally, from clinically, as a clinician, was very exciting to see the meaningful, robust, clinical improvement. So all the four patients were able to achieve normal level of hemoglobin by month number three. And with even this short number and short follow-up, we were seeing elevation in the fetal hemoglobin above 40%, which is really helping to decrease or prevent any sickle cell and any complication. So now novel things about EDIT-301. It’s really using, as I mentioned, the Caspase 12, which is a new using the CRISPR technology, but the Caspase is a single RNA, so it has some advantage in terms of higher efficiency. But the second thing that is unique about it, it is targeting directly the hemoglobin G1 and G2 promoter. So we’re looking forward to enroll the rest of the patients and be able to finish the two-year follow-up and give an update for the study later.