FDA Authorizes Clinical Trial for CRISPR/Cas9 Therapy for LCA 10
Editas Medicine , a company developing gene-editing treatments, has received authorization from the US Food and Drug Administration to launch a clinical trial for its emerging CRISPR/Cas9 therapy for people with a mutation in the gene CEP290, which causes Leber congenital amaurosis 10 (LCA10). LCA causes severe vision loss or blindness at birth. Known as EDIT-101, the treatment will be evaluated in a Phase 1/2, dose-escalation clinical trial. The company expects to enroll 10 to 20 patients. Further details and contact information for the study will be listed soon at www.clinicaltrials.gov . EDIT-101 targets a specific mutation, “c.2991+1655A>G” in intron 26, of the CEP290 gene. Editas has earned a $25 million milestone payment from Allergan as part of a drug-development alliance. “We are very pleased with this historic milestone in the treatment of inherited retinal diseases,” says Stephen Rose, PhD, chief scientific officer, Foundation Fighting Blindness. “EDIT-101 is the fi...
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