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Foundation Fighting Blindness Grants RetroSense Therapeutics $250,000 for Research into Using Genes from Algae to Reverse Severe Vision Loss

Columbia, MD (February 10, 2012) – The Foundation Fighting Blindness, a national nonprofit dedicated to driving research for retinal degenerative diseases, announces a $250,000 research grant to Ann Arbor, Mich.-based RetroSense Therapeutics for developing a treatment that delivers the genes of blue-green algae to the retina for restoring vision. The funding supports RetroSense’s lab research in preparation for a clinical trial of the gene therapy in two to four years.

“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.


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