The FDA has accepted an investigational new drug application for Editas Medicine’s experimental CRISPR genome editing medicine for the treatment of Leber congenital amaurosis type 10, the company announced in a press release.
EDIT-101 is administered as a subretinal injection to deliver gene editing machinery to photoreceptor cells.
“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,” Editas President and CEO Katrine Bosley said in the release.
The acceptance sparks a $25 million milestone payment to Editas from Allergan as part of a partnership between to two companies to develop experimental ocular medicines.
A phase 1/2 open-label, dose-escalation study of EDIT-101 is planned to evaluate its safety, tolerability and efficacy.