Editas' Gene Editing Treatment Shows Initial Safety, Efficacy In Patients With Rare Blood Disorders

Editas Medicine Inc EDIT announced initial safety and efficacy data from the first four patients with sickle cell disease (SCD) treated with EDIT-301 in the RUBY trial and from the first transfusion-dependent beta-thalassemia patient treated in the EdiTHAL trial.

Patient 1’s total hemoglobin returned to a normal physiological level of 16.4g/dL at five months after infusion of EDIT-301 and has been maintained at this level at the 10-month follow-up. 

In addition, Patient 1’s fetal hemoglobin fraction increased from 5% at baseline to 45.4% five months after treatment with EDIT-301 and 43.4% at the 10-month follow-up.

Patient 2’s total hemoglobin reached a normal physiological level of 12.7 g/dL at five months after infusion of EDIT-301, and fetal hemoglobin increased from 10.8% at baseline to 51.3% at the 6-month follow-up.

Patients 3 and 4 in the RUBY trial saw total and fetal hemoglobin increases at three and two months of follow-up, respectively.

All four treated RUBY patients are also free of vaso-occlusive events (VOEs) since infusion.

In the EdiTHAL trial, the first patient demonstrated successful neutrophil and platelet engraftment, and, at one and a half months post-infusion, the patient’s response resembles that of the first four RUBY patients.

The first EdiTHAL patient’s experience with EDIT-301 resembles that of the first four RUBY patients, achieving a fetal hemoglobin fraction of 34.9%, representing 4 g/dL of total hemoglobin at 1.5 months post-treatment.

EDIT-301 was well-tolerated and demonstrated a safety profile. 

After EDIT-301 infusion, no serious adverse events occurred, and no adverse events reported were related to treatment with EDIT-301.

Price Action: EDIT shares are down 1.51% at $9.46 on the last check Friday.