Success Story: New Gene Therapy for Dry AMD

With support from MLERFi, researchers at Tufts University School of Medicine have developed a new gene therapy that will be tested in human patients In 2016. Specifically, this therapy will target the ‘dry’ form of AMD that currently accounts for 90% of AMD patients, none of whom can be currently treated. This gene therapy uses a harmless virus to deliver a therapeutic gene (that was developed in the laboratory) to the eyes of AMD patients. The researchers have also made significant progress in the development of gene therapies for Retinitis Pigmentosa and Diabetic Retinopathy but these are not yet at the stage of being ready for testing in patients. If successful, we will have the potential to positively impact the lives of millions of people worldwide.