RetroSense, WSU sign license agreement for channelrhodopsin-based approaches

Oct 19 2011

RetroSense Therapeutics, LLC, a Michigan-based company, announced that it has executed its exclusive, worldwide option and signed a license agreement for novel gene-therapy approaches for treating blindness developed at Wayne State University's School of Medicine.

Zhuo-Hua Pan, Ph.D., professor of anatomy and cell biology in the School of Medicine, along with colleagues at Salus University in Pennsylvania, developed the breakthrough therapy and follow-on approaches that offer promise to people suffering with incurable 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 people in the U.S. alone. Worldwide, 500,000 individuals lose their eyesight annually to AMD, which is the result of progressive deterioration of photoreceptor cells in the macula, the central portion of the retina.

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

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), Pan found that ChR2 made the inner retinal neurons become light sensitive, and that it persisted for long periods in the neurons, ultimately leading to restored responses to light in the visual cortex of the brain.

With this technology, combined with the business and drug development expertise of RetroSense's seasoned management team, Pan is hopeful his breakthrough treatment is on the fast track to restoring a vital part of the human experience to people suffering from retinal degeneration worldwide.

"Our research has demonstrated the feasibility of restoring visual responses after the loss of light-sensitive photoreceptor cells in animal models," said Pan. "The ChR2-based gene therapy is a promising strategy for treatment of blindness caused by the death of photoreceptor cells in humans."

"We are delighted to enhance our position in this space by licensing these novel approaches to vision restoration," said Sean Ainsworth, founder and CEO of RetroSense. "Channelrhodopsin-based approaches to vision restoration are garnering a great deal of attention from academia and industry right now. It's a very hot field and we are quite pleased to be aligned with Dr. Pan and his colleagues who pioneered the approach."

Ainsworth licensed the technology from Wayne State University in an effort to eventually test the technology in humans. While there are several years of research and testing needed before anything can be brought to the market, Phase I clinical trials could begin in early 2013.

"This license agreement with RetroSense is an exciting example of how critical research is to making discoveries, then getting those discoveries to the market," said Hilary Ratner, vice president for research at Wayne State University. "Life-changing developments often happen because of university research, and relationships with companies such as RetroSense are critical components of taking a faculty member's ideas and inventions to the next level for creating commercially viable products and processes. We are pleased to welcome RetroSense to the Wayne State University community."

Source: Wayne State University - Office of the Vice President for Research