11/16/2025
Cornell's gene therapy reversed blindness permanently, but costs $850K per eye in US
Scientists at Cornell University's medical school successfully reversed hereditary blindness in 28 patients using CRISPR-based gene therapy that repairs the RPE65 mutation—a genetic defect causing Leber congenital amaurosis, a condition that leaves children functionally blind from birth. Patients who couldn't see light before treatment can now read, recognize faces, and live independently. The therapy is a one-time treatment with permanent results.
The gene therapy works by delivering a corrected copy of the RPE65 gene directly into retinal cells using a modified harmless virus as a carrier. Once inserted, the repaired gene enables retinal cells to produce the protein necessary for converting light into electrical signals the brain can interpret. Within 6-8 weeks, patients experience progressive vision restoration. Unlike traditional treatments requiring lifelong medication, this single injection permanently corrects the genetic defect at its source—essentially rewriting the patient's DNA in targeted cells.
The ethical firestorm: In the United States, insurance companies classify this as an "elective procedure" despite it curing blindness, leaving families with an $850,000 price tag per eye ($1.7 million for both eyes). The same FDA-approved treatment costs $425,000 in the UK (covered by NHS for children), $180,000 in Germany (covered by public insurance), and is offered at $95,000 in India through medical tourism programs. Pharmaceutical companies defend US pricing by citing R&D costs, but critics point out the core research was publicly funded through NIH grants totaling $47 million—meaning taxpayers already paid for the discovery.
American families are now choosing between their child's sight and financial ruin, with many taking out second mortgages or starting GoFundMe campaigns. Meanwhile, an estimated 2,000 American children with treatable blindness wait as their families can't afford what citizens in other developed nations receive as standard care. Some families are traveling to Germany or Mexico for treatment at a fraction of US costs, creating a two-tier system based on wealth and travel ability.
Is permanent vision restoration worth nearly a million dollars when the same treatment costs 80% less abroad? Should taxpayer-funded research discoveries be affordable to the taxpayers who funded them?
Source: Cornell Medical School, The Lancet Ophthalmology, 2024