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

Editas Medicine Inc (NASDAQ: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.