Editas Medicine announced that the FDA has accepted the company’s investigational new drug (IND) application for EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of Leber Congenital Amaurosis type 10 (LCA10).
“The FDA’s acceptance of our IND for EDIT-101 is a significant moment in the field of genome editing, and importantly, a critical milestone for patients, as we are now one step closer to a treatment for LCA10,” Katrine Bosley, President and Chief Executive Officer, Editas Medicine, said in a company news release. “This moment is a truly exciting one for us, and we look forward to embarking on our next chapter as a clinical stage company, harnessing the power of CRISPR technology to transform the lives of people with serious diseases around the world.”
With the IND acceptance, Editas Medicine has earned a $25 million milestone payment from Allergan as part of the alliance between the companies to discover and develop experimental ocular medicines targeting serious, vision-threatening diseases. Editas Medicine and its partner Allergan expect to enroll 10 to 20 patients in a phase 1/2 open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101.