Gene therapy for blindness gets attention of investors

Ann Arbor-based RetroSense Therapeutics LLC plans to start human trials of a gene-based therapy next year, and if those trials go as animal trials have gone, those blinded by degenerative eye diseases will regain some of their vision.

What seemed more like an interesting science experiment four years ago than a real company -- can a light-sensing gene that helps pond scum find sunlight also help the blind see? -- is attracting interest and venture capital as it gears up for human trials.

The therapy is based on the photosensitivity of a gene called channelrhodopsin-2. This gene allows blue-green algae to detect where the sun is shining on a pond so they can move in its direction and convert light to energy through photosynthesis.

When the gene, which is inside a cultured medium called a vector, is injected into the eye, previously non-photosensitive retinal cells are converted into photosensitive cells, allowing limited vision. RetroSense and its research partners have tested mice, rats and nonhuman primates, with all species showing a return of some vision following treatment, said founder and CEO Sean Ainsworth.

As a result, the company has just completed raising a venture capital round of $2.4 million, led by a San Diego firm, Nerveda LLC, and is close to agreeing on terms of a follow-on round of $5 million or more, Ainsworth said.

Those rounds will fund Phase 1 U.S. Food and Drug Administration trials next year and the first half of Phase 2 trials.

Fundraising has been sparked because of large recent deals involving other biotech firms and by RetroSense's success in animal trials for its lead product, a biologic with the working name of RST-001.

RetroSense CEO Sean Ainsworth

Ainsworth founded RetroSense in 2010 after licensing the work of Wayne State University researcher Zhuo-Hua Pan. He laid out the company's plans to a standing-room-only crowd at the recent Michigan Growth Capital Symposium in Ypsilanti, which drew venture capitalists and angel investors from around the country to hear pitches for capital from 32 Midwest firms.

Ainsworth said that if what has worked in animals works in patients taking part in the upcoming trials, at least some black-and-white vision will be restored to those blinded by such diseases as retinitis pigmentosa and dry age-related macular degeneration.

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Gene therapy for blindness gets attention of investors