Why is Gene Therapy Exciting?

Why is gene therapy exciting? First, it provides treatments for diseases that we could never treat before such as genetic eye disease also known as inherited retinal degenerations (IRDs). It treats genetic disease at its root cause. Gene therapy is durable, it can last a long time. How long exactly is unknown, now but at least several years. Lastly gene therapy bridges medical unmet needs with the wonderfully amazing world of modern molecular biology!

Theodore Friedmann and Richard Roblin introduced the concept, ethical considerations, and scientific unknowns regarding gene therapy for human disease in their article in Science magazine published in 1972. Forty-Five years later in 2017, the FDA approved the first gene therapy for a human inherited retinal disease voretigene neparvovec-rzyl, used to treat biallelic RPE65-associated retinal dystrophy. The early science behind voretigene neparvovec-rzyl started at the University of Pennsylvania, led by Dr. Jean Bennett and Dr. Al Maguire. The field of ophthalmic genetics owes much to the visionary and translational work done by Dr. Bennett and Dr. Maguire. The success of voretigene neparvovec-rzyl therapy has generated an avalanche of research into new gene therapies for eye disease. 

At Genetic Eye Therapies we are forward looking with regards to the use of gene therapy for human eye disease. Importantly, we also understand that advances need to be grounded in scientific rigor, patient centered ethics, and well vetted technology and clinical trials.