The FDA has approved Luxturna, an innovative one-time gene therapy treatment from Spark Therapeutics used to improve vision in patients with vision loss due to inherited retinal diseases (IRD). As the first adeno-associated viral (AAV) vector product approved for clinical use in the United States, Luxturna represents a historic landmark, not only in the field of gene therapy, but in the history of medicine and opens the door as a proven viral vector product for the many AAV products currently under development and in clinical trials throughout the country.
“Having the first approval of a gene vector delivered directly to a person sets a precedent for other groups to move forward to develop gene therapies to treat other inherited and acquired diseases,” Dr. Jean Bennett, F.M. Kirby Professor of Ophthalmology at the University of Pennsylvania’s Perelman School of Medicine and a leader in the development of Luxturna, says. “Until now, there has been no path.”
“There are going to be a lot more approaches using AAV coming down the line both in eye gene therapy and in other diseases such as hemophilia,” said Dr. Helen E. Heslop, American Society of Gene & Cell Therapy (ASGCT) President and Director of the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital. (Source: American Society of Gene & Cell Therapy Website, December 19, 2017)