Gene Therapy in Ophthalmology Update 2: Foundation Fighting Blindness Funds Six New Gene Therapy Projects
Posted Oct 31 2011 4:39pm
In a news release that I found on the net, I learned that the FFB was going to put $8.25 million into six gene therapy projects, either already underway or about to start. The release contains good information about several projects that I knew about, and others that I did not.
The Foundation Fighting Blindness, a national nonprofit dedicated to advancing sight-saving research, announces an $8.25 million investment in six new gene therapy research projects that are targeted to have treatments ready for clinical trials within three years. The grants focus on treating a broad range of retinal degenerative diseases and will be allocated through the Foundation's Translational Research Acceleration Program, which funds research efforts with strong, near-term clinical potential.
"The Foundation Fighting Blindness recognizes the great potential of gene therapy for saving and restoring vision, and we're eager to build on the clinical development of retinal gene therapies that has been accelerating at an incredible rate over the past few years," said Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. "It was just three years ago that we reported groundbreaking results from our first gene therapy clinical trials that restored vision in children and young adults who were virtually blind from Leber congenital amaurosis (LCA). The success of those studies set the stage for this rapid expansion in gene therapy development."
As part of the new $8.25 million investment, one innovative project involves the use of gene therapy to resurrect and reactivate cone cells that are compromised by disease. In many inherited retinal conditions, including retinitis pigmentosa, cones stop working before they completely degenerate. The Institut de la Vision in Paris and the Friedrich Miescher Institute in Basel, Switzerland, are developing a gene therapy that revives degenerating cones, enabling them to regain their ability to respond to light and provide vision. The treatment also improves the health of cones and extends 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 can improve an affected individual's well being, because these cells provide central, daytime and detailed vision that is critical for independent living.
The Foundation is also funding the Oklahoma University Health Sciences Center, which in collaboration with Copernicus Therapeutics, is developing a nanoparticle gene therapy system. Nanoparticles are tiny manmade particles, 1/12,000th the diameter of a human hair, which can readily penetrate retinal cells, 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 ability to deliver large genes - genes that exceed the capacity of viral delivery systems - for treating some diseases.
Through a Foundation grant to Applied Genetic Technologies Corp. (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 United States and Europe.
Portions of the Foundation's $8.25 million investment will also go toward research happening at the Massachusetts Eye and Ear Infirmary and the University of Florida for projects investigating gene therapy for two different LCA-causing genes. The final grant supports work at the University of Pennsylvaniafor 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.
There are now human studies of gene therapy underway for Leber congenital amaurosis, wet age-related macular degeneration, and Stargardt disease, with clinical trials for Usher syndrome (the leading cause of deaf-blindness) type 1B and autosomal recessive retinitis pigmentosa scheduled to begin in late 2011 or early 2012. Currently supporting 30 other gene therapy efforts, including RDH12 and other genetic forms of LCA and RP, which are at various stages of development, the Foundation allocates funding toward basic research and investigation into a gene's role in disease, as well as projects poised for clinical trials.
Editor's Note: I have put together a matrix, that at the moment contains eight companies involved in Gene Therapy in Ophthalmology projects. Anyone wishing to obtain a copy of the matrix, please email me.