ASH 2024 | Updated results of RUBY: the positive effects of reni-cel gene therapy in patients with severe SCD

I’m very excited to give an update during ASH, and I would encourage everyone to come and visit us at the poster. So we are giving an update for the RUBY trial, which is using gene editing technology with Cas12, but it differs from the other technologies that are already approved by targeting hemoglobin G1 and G2. 

And in ASH 2024, we’re giving an update on 28 patients now. So that’s for the safety profile for them has been consistent with all the previous reports. No patient has died. We have two patients who have adverse effects related to the conditioning therapy with busulfan, and that manifested in a patient with severe ARDS and also gastritis. 

And we are presenting an update about the efficacy in 27 patients, and the data show consistency with the previous report where the patient engrafted their neutrophils in 23 days, and the platelet engraftment was around 25 days. But what is so exciting, even in this expanded cohort, we have consistency and robust clinical efficacy where only one patient has severe pain crisis that was related and in the settings of an admission for COVID infection, and that patient of note had 21 pain crises in the prior two years prior to the enrollment. 

So, in summary, I think this expanded cohort shows persistently higher levels of total hemoglobin and the pan expression of the fetal hemoglobin. We show robust clinical improvement in the pain crisis, which is the primary endpoint with only one patient out of this 27 that had a pain crisis that was actually in the setting of an infection. The safety profile of this patient is consistent with autologous with no death reported in this expanded cohort.

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