Editas Medicine is on track to “make history” with the enrollment of its in vivo CRISPR genome-editing technology clinical trial, CEO Cynthia Collins said at the J.P. Morgan Healthcare Conference.
“We are entering 2020 with strong momentum and a strategic focus on driving our pipeline of in vivo CRISPR and engineered cell medicines forward with the ultimate vision of developing differentiated, transformational medicines for people living with serious diseases,” Collins said.
EDIT-101, which is targeting genetic blindness, removes the genetic mutation that causes degeneration of photoreceptors in order to restore the CEP290 protein and rebuild photoreceptors in patients with Leber congenital amaurosis 10. The CEP290 protein is essential to photoreceptor function, in particular the outer segment that senses light. The “editing” process aims to correct the splicing defect in the gene that encodes the CEP290 protein, which will regenerate the outer segment, restoring light sensitivity with hopes that it will also restore sight.
The phase 1/2 open-label, dose-escalation Brilliance clinical trial will evaluate safety, tolerability and efficacy in 18 subjects dosed via subretinal injection. Doses will be on the lower end of the therapeutic range in adults with only light perception and will escalate based on safety and tolerability; the study will eventually move into children with some visual acuity. Data may be reported by the end of the year.
“We are making progress on both pillars of our therapeutic strategy to create differentiated transformational medicines for diseases of high unmet need. For our in vivo medicines, we are advancing EDIT-101, the first-ever in vivo CRISPR medicine, in the clinic,” Collins said. – by Kate Burba
Disclosure: Collins reports she is CEO of Editas Medicine.