CRISPR Base Editing Therapy Approved: Hereditary Blindness Patients Regain Sight

On January 17, 2028, the US FDA approved Editas Medicine's CRISPR base editing gene therapy Edit-102 for treating Leber congenital amaurosis type 10 (LCA10). This is the world's first approved base editing gene therapy and the second approved CRISPR therapy after Casgevy in 2023.

Unlike traditional CRISPR-Cas9, base editing technology doesn't cut DNA double strands — it directly "erases and rewrites" one base into another. For LCA10, caused by a single base mutation, base editing can precisely repair the mutation site while avoiding off-target risks from DNA double-strand breaks.

Phase III clinical trial data shows that among 48 treated patients, 83% improved by more than two grades on standard visual acuity charts at 12 months post-injection, with 41% improving by more than four grades. Three patients who were completely blind before treatment regained independent indoor navigation ability.

Editas Medicine CEO Gilmore O'Neill stated: "Edit-102's approval marks gene editing's transition from the cutting era to the erasing era. Base editing's precision allows us to safely repair single-base mutation genetic diseases."

The therapy is priced at $420,000 per eye, totaling approximately $840,000 for bilateral treatment. Editas has negotiated installment payment agreements with multiple insurers. FDA also requires Editas to conduct 15 years of long-term follow-up to monitor gene editing safety.

Harvard's David Liu laboratory invented base editing technology. Professor Liu commented that Edit-102's approval will accelerate clinical development of other base editing therapies, with over 30 base editing projects currently in clinical or preclinical stages.