At long last, Editas Medicine has made it. The gene-editing biotech and its partner, Allergan, are moving their treatment for a rare form of blindness into human testing. Though they are not the first companies to test a CRISPR-based medicine in humans—that distinction goes to CRISPR Therapeutics and Vertex Pharmaceuticals—their treatment is the first that edits DNA within the body.
The duo is developing a treatment for Leber congenital amaurosis 10, or LCA10, a type of blindness caused by mutations in the CEP290 gene. They had once expected to file the IND for the treatment, first dubbed EDIT-101 or AGN-151587, by the end of 2017, but manufacturing issues delayed that timeline to October 2018.
The FDA signed off on the phase 1/2 in November, and the partners have started enrolling patients in the U.S. The Brilliance study will test three doses of the gene-editing treatment in up to 18 children and adults with LCA10. After undergoing vitrectomy—a procedure that removes some of the gel-like tissue in the eye—patients will receive one injection of AGN-151587 behind their retina. The treatment, which gets rid of the mutation that causes LCA10, is delivered via adeno-associated virus (AAV) directly to the eye’s light-sensing photoreceptor cells.
“Now that enrollment is underway, we are one step closer to delivering a transformative medicine to LCA10 patients,” said Charles Albright, Ph.D., chief scientific officer at Editas. “The team at Editas looks forward to continuing to collaborate with our partners at Allergan, patient advocacy organizations, and the inherited retinal diseases community as we develop this and other durable experimental medicines for patients with devastating ocular diseases.”
While Editas and Allergan’s treatment edits the CEP290 gene inside the body, CRISPR Therapeutics and Vertex’s treatment does it outside the body. They kicked off a phase 1/2 study of their beta thalassemia treatment in Germany last August. Although research groups in China had already begun testing CRISPR in humans, CRISPR Therapeutics and Vertex were the first companies to sponsor a human trial of the gene-editing technology.
They are developing the treatment, CTX001, for sickle cell disease as well as beta thalassemia, both of which are caused by mutations in the beta-globin gene, which codes for a part of hemoglobin, the oxygen-carrying component of red blood cells. This causes patients to make faulty hemoglobin—or none at all.
CTX001 is an autologous, gene-edited hematopoietic stem cell therapy, meaning it is made of a patient’s own blood cells. The cells are harvested from the patient, edited to increase fetal hemoglobin levels and then infused back into the patient, where they are expected to produce blood cells with fetal hemoglobin and compensate for defective adult hemoglobin.
While Editas was playing catch-up, it lost two top execs. In August, the company announced Chief Medical Officer Gerald Cox, M.D., would be departing at the end of the year. Barely three weeks after his exit, CEO Katrine Bosley said she was calling it quits by March 1, with board member Cynthia Collins stepping up to the plate as Editas seeks a successor.