EDIT-101

EDIT-101 is an investigational gene therapy that uses a recombinant adeno-associated virus serotype 5 (AAV5) vector to deliver CRISPR/Cas9 gene editing machinery directly to photoreceptor cells in the retina. The therapy is designed to treat Leber congenital amaurosis type 10 (LCA10), a severe inherited retinal degenerative disorder caused by mutations in the CEP290 gene, specifically the c.2991+1655A>G (IVS26) mutation. By delivering Staphylococcus aureus Cas9 and two guide RNAs, EDIT-101 enables targeted excision or inversion of the IVS26 mutation, aiming to restore normal CEP290 protein expression and improve photoreceptor function. It is administered via subretinal injection and has received Rare Pediatric Disease and Orphan Drug designations from both the FDA and EMA[1][4][5][6][8].

Other names

AGN-151587AGN151587AGN 151587