Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.
Editas CMO Baisong Mei and CEO Gilmore O’Neill/courtesy of Editas Medicine
Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.
EDIT-101 is in development for a rare genetic eye disorder, Leber congenital amaurosis 10 (LCA10), which is the most common cause of inherited blindness in childhood. In a Phase I/II BRILLIANCE trial, the in vivo CRISPR/Cas9 editor provided clinically meaningful improvements to over 20% of patients. However, the trial population was relatively small, translating to three patients out of fourteen.
Two of the three patients who experienced the benefit were homozygous, meaning they have two copies of the IVS26 mutation. The data suggest patients with this mutation would benefit the most from this treatment. The patient population with this mutation is about 1 out of 5 LCA cases.
"[This totals] about 300 patients in the United States, which for our business model, would not be viable,” CEO Gilmore O’Neill told BioSpace in an interview.
“We’re very sensitive to patients and so we’re looking for a partner who would have an interest in genetic therapies for rare diseases or diseases with the necessary expertise but also with a business model that supports an ultra-rare population.”
O’Neill was brought on as CEO in June to help usher in a new era for Editas. Between himself and his chosen CMO, Baisong Mei, the two have brought 10 drugs through to the market. That’s the hope O’Neill has for Editas too.
“We are going to bring Editas from a therapeutic technology platform company into a commercial therapeutics company,” he said. “That is my key vision, our shared vision of the organization. It’s the right time now for us.”
O’Neill kept a tight lid on any partners under consideration for both EDIT-101 and the company’s oncology line. Editas is rumored to be in “advanced discussions” regarding a potential sale of its oncology assets.
He said Editas is seeking a partner for the EDIT-301 program for commercial and development outside the U.S. This is the company’s most advanced program, an ex vivo gene-edited medicine in clinical trials for sickle cell disease and transfusion-dependent beta-thalassemia. Data from the Phase I/II RUBY trial will be presented next month.
