In Nature, Luk Vandenberghe, PhD, Harvard Medical School Associate Professor of Ophthalmology and Director of the Grousbeck Gene Therapy Center at Mass. Eye and Ear, discusses increasing the efficiency of gene therapies by manipulating the viral vectors that transport genes into cells. He also talks about taking a nonprofit approach to tackle the economics of developing gene therapy for rare diseases.
Dr. Vandenberghe’s research focuses on gene therapy for neurosensory disorders, especially retinal blindness. In 2011, he cofounded GenSight Biologics, a biotechnology company that pursues innovative ocular gene therapy strategies. Most recently, he cofounded Odylia Therapeutics, a nonprofit corporation dedicated to creating gene therapies for ultra-rare genetic conditions that cause blindness.