Foundation Fighting Blindness Grants RetroSense Therapeutics $250,000 for Research into Using Genes from Algae to Reverse Severe Vision Loss
Published: Feb 10, 2012
“Imagine if the secret to reversing blindness lies in the DNA of pond scum,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “This field of research, known as optogenetics, is a very promising and creative approach to restoring sight in people with severe vision loss or complete blindness due to inherited retinal diseases. RetroSense has outstanding technology, and it has achieved impressive results in lab studies. We are pleased to support the company in its effort to move the treatment into a clinical trial.”
In initial pre-clinical work, RetroSense has demonstrated that its treatment restores vision in mice with highly degenerated retinas. The current funding will help advance the initial studies toward a stage where the company could seek authorization for a Phase I clinical trial to confirm the therapy’s safety. The genetic treatment is delivered to retinal ganglion cells, which survive long after rods and cones, the cells that normally provide vision, are lost to advanced retinal degenerations like retinitis pigmentosa (RP). The treatment contains copies of an algal gene called channelrhodospin-2, which makes the ganglion cells light-sensitive. Normally, ganglion cells don’t provide vision; rather they help fine-tune the visual information generated by rods and cones.
“One big advantage of optogenetic treatments, including RetroSense’s, is that, because they are independent of the genetic defects causing disease, they will likely work for a variety of retinal degenerations,” says Dr. Rose. “It is exciting to support a therapeutic approach that has the potential to benefit many people.”
The company’s gene therapy uses an adeno-associated virus, or AAV, for delivering the therapeutic gene to the cells of the retina. The technology has performed well thus far in vision-restoring clinical trials of gene therapy for people with Leber congenital amaurosis. More than 40 people have been successfully treated in those studies.
The Foundation is also funding an optogenetic therapy project to revive cone cells — the cells in the retina that provide color and central vision. That effort is being led by Dr. José-Alain Sahel, director of the Foundation-funded Paris Research Center for the Study of Retina Degenerative Diseases, and Dr. Botond Roska of the Friedrich Miescher Institute for Biomedical Research in Switzerland.
About the Foundation Fighting Blindness
The Foundation Fighting Blindness is a national nonprofit driving research that will lead to preventions, treatments and cures for retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases that affect more 10 million Americans. In its 40-year history, the Foundation has raised more than $450 million as the leading non-governmental funder of retinal research. Breakthrough Foundation-funded studies using gene therapy have restored significant vision in children and young adults who were previously blind, paving the way for using this method to treat a wide variety of retinal degenerative diseases, and proving a cure is in sight. With a network of nearly 50 chapters, the Foundation also provides support and resources to affected individuals and their families in communities across the country.