WSU's novel gene-therapy for blindness receives $250,000 FFB grant

Technology to restore vision through the use of a component of green algae - developed by a Wayne State University professor and scientific director of the Ligon Research Center of Vision at the Kresge Eye Institute - has attracted additional funding for therapy development.

The Foundation Fighting Blindness announced a $250,000 grant from their affiliate, National Neurovision Research Institute, to RetroSense Therapeutics, LLC, a Michigan-based company. RetroSense signed a license agreement in 2011 for the novel gene-therapy approaches developed at Wayne State University by Zhuo-Hua Pan, Ph.D., professor of ophthalmology and anatomy & cell biology in the School of Medicine.

Pan's novel strategy focused on genetically converting light-insensitive inner retinal neurons into photosensitive cells, thus restoring light sensitivity to retinas that lack photoreceptors. Using a virus that delivers a photoreceptor gene from green algae called channelrhodopsin-2 (ChR2), he found that ChR2 made the inner retinal neurons light sensitive, and that this persisted for long periods in the neurons, ultimately leading to restored responses to light in the brain's visual cortex.

Pan, along with colleagues at Salus University in Pennsylvania, developed the breakthrough therapy and follow-on approaches that offer promise to people suffering with blindness caused by age-related macular degeneration (AMD) and retinitis pigmentosa (RP) - retinal degenerative disorders that are currently incurable.

AMD is the leading cause of blindness in people older than 60, affecting more than 8 million in the United States. Worldwide, 500,000 individuals lose their eyesight annually to AMD, which is the result of progressive deterioration of photoreceptor cells in the macula, near the center of the retina.

RP is a genetically-determined eye disease caused by mutations in more than 100 different genes. An estimated 100,000 Americans have RP, which typically manifests as night blindness and progresses to tunnel vision and sometimes complete blindness.

"This field of research, known as optogenetics, is a very promising and creative approach to restoring vision in people with severe vision loss or complete blindness," said Stephen Rose, M.D., the foundation's chief research officer, in a news release. "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."

Rose said an advantage of optogenetic treatments is that because they work independently of genetic defects causing disease, they may work for a variety of retinal degenerations.

The foundation said the therapy developed by Pan has demonstrated restored vision in animal models with highly degenerated retinas. RetroSense Therapeutics is seeking U.S. Food and Drug Administration approval for a Phase I clinical trial.

Sean Ainsworth, founder and chief executive officer of RetroSense, licensed the technology from Wayne State University. He has said that Phase I clinical trials could begin in early 2013.