Recently, the Foundation Fighting Blindness announced an $8.25 million investment in six new gene therapy research projects targeted to have treatments ready for clinical trials within three years. The projects include the following:
- The Institut de la Vision in Paris and the Friedrich Miescher Institute in Basel, Switzerland, are developing a gene therapy that may revive degenerating cones and potentially enable them to regain their ability to respond to light and provide vision. The treatment also has the potential to improve the health of cones and extend their lifespan significantly. This therapeutic approach holds the potential to benefit people affected by a range of conditions, because it works independently of the underlying disease-causing genetic defect. Resurrecting cones may be able to improve an affected individual’s well being, because these cells provide central, daytime and detailed vision that is critical for independent living.
- The Oklahoma University Health Sciences Center, which in collaboration with Copernicus Therapeutics, is developing a nanoparticle gene therapy delivery system. Nanoparticles are tiny manmade particles, 1/12,000th the diameter of a human hair, which can potentially penetrate retinal cells, possibly making them effective for delivery of therapeutic genes. They may provide advantages in certain cases over viral gene delivery technologies currently used in retinal disease therapies. Perhaps most beneficial is their potential to deliver large genes — genes that exceed the capacity of viral delivery systems — for treating some diseases.
- AGTC, a clinical stage biotechnology company, funds will support researchers at Oregon Health & Science University’s Casey Eye Institute and the University of Florida in their pre-clinical work to evaluate a gene therapy treatment for X-linked retinoschisis, a blinding disease that affects over 35,000 patients in the U.S. and Europe.
- The Massachusetts Eye and Ear Infirmary and the University of Florida are investigating gene therapy for two different LCA-causing genes.
- The University of Pennsylvania is working on choroideremia gene therapy led by Dr. Jean Bennett, who is also one of the lead investigators on the landmark LCA gene therapy clinical trial that has restored vision in more than 40 patients.
The Foundation Fighting Blindness is a national nonprofit dedicated to advancing sight-saving research. The Foundation funds research toward preventions, treatments and cures for vision-robbing retinal degenerative diseases including macular degeneration, retinitis pigmentosa, Usher syndrome, and several other rare conditions. These newly-funded projects build on the success of landmark gene therapy clinical trials now underway that have restored some vision in children and young adults who were virtually blind.