EDIT - Editas posts initial data for gene editing candidate for blood disorders

2023-06-09 10:41:41 ET

Editas Medicine ( NASDAQ: EDIT ) on Friday announced initial safety and efficacy data from a pair of trials designed to evaluate EDIT-301, a gene editing candidate targeted at inherited blood disorders, sickle cell disease (SCD), and beta-thalassemia.

Citing data from the first four patients in the company’s Phase 1/2 RUBY trial, Editas ( EDIT ) said two patients on EDIT-301 achieved normal hemoglobin levels five months after starting the treatment, keeping normal hemoglobin levels at ten- and six-month follow-up.

The company said the other two patients had seen rising total hemoglobin and fetal hemoglobin at three and two months of follow-up, indicating the pattern in the first two patients. All four patients remained free of vaso-occlusive events since EDIT-301 therapy.

Disclosing data from Editas’ ( EDIT ) Phase 1/2 EdiTHAL trial for transfusion-dependent beta-thalassemia, the company said the first patient to receive EDIT-301 indicated a response similar to that seen in the first four patients in the RUBY trial.

The company also noted that there were no serious adverse events post EDIT-301 infusion, and none of the adverse events were related to the experimental therapy.

RUBY data will be presented at the Annual European Hematology Association (EHA) Congress on Saturday.

More on Editas

• Editas Medicine appoints Erick Lucera as CFO, Michelle Robertson steps down
• Editas Medicine: Pioneering Gene-Editing Solutions For Inherited Blood Disorders And Beyond

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