Following a management reshuffle, Editas Médecine received some interesting news. Biotechnology received a second rare pediatric disease designation from the FDA for its genetically modified drug EDIT-301.
EDIT-301 has already been approved for the treatment of sickle cell anemia and is currently being evaluated in a clinical trial. Following recent FDA approval, Editas plans to initiate a Phase 1/2 study of EDIT-301 in patients with transfusion-dependent beta-thalassemia this year.
Gene editing biotechnology has been in recent news, but not necessarily because of its clinical treatments. The company announced a series of leadership changes, leading Gilmore O’Neill, Sarepta’s chief marketing officer, to replace president and CEO James Mullen, who will serve as executive chairman of Editas starting June 1. Interestingly, O’Neill does not hold the position of medical director of biotechnology, which remains vacant following the expulsion of the company Lisa Michaels, MD, in February with no explanation.
The company is also embroiled in an ongoing patent battle, where victory came in February when the U.S. Patent and Trademark Office ruled in favor of Harvard and the MIT Broad Institute in a related patent dispute. to the CRISPR gene-editing technology. Editas holds patents with this group and the share price has risen significantly following this decision. However, the opposition group appealed to the United States. The Federal Circuit Court of Appeals eventually dropped the case.
The company announced its first test data last fall, but the limited data failed to impress investors. The latest FDA label for beta-thalassemia could help bolster the company’s case history for its gene-editing therapies.
“Beta-thalassemia is a devastating disease that leads to severe anemia, organ failure, and premature death,” Mullen said in an April 26 news release. “EDIT-301 is a potentially transformative drug for patients with beta-thalassemia and we look forward to rolling out the first patient in our clinical trial this year.”