IND application accepted for CRISPR genome editing treatment for LCA10

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.

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.