US accepts Editas’ IND for experimental CRISPR medicine

Gene editing with CRISPR used to treat lethal lung diseases in utero
Credit: Gek

Cambridge, Massachusetts-based genome editing company Editas Medicine has had its Investigational New Drug application for EDIT-101, an experimental CRISPR genome editing medicine, approved by the FDA.

EDIT-101 is currently 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,” said Katrine Bosley, President and CEO of Editas Medicine.

“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 Pharmaceuticals International Limited, 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.