Startups, BioPharma

Restoring vision with gene therapy: RetroSense gets $6M

RetroSense Therapeutics, an Ann Arbor optogenetics startup, is developing an algae-based gene therapy that could restore functional sight in those with congenital vision loss disorders.

An Ann Arbor optogenetics startup just raised capital to fuel clinical research for its gene therapy that could help restore vision.

RetroSense Therapeutics just closed out a $6 million Series B round to help the company complete a Phase 1/2 clinical study of its lead compound, RST-001, to treat retinitis pigmentosa. The funding will also help RetroSense develop a second clinical candidate.

This round is right on the heels of an angel-backed $7 million Series A earlier this year. The Wayne State University spinout was honored earlier this year as the most innovative startup at the Angel Capital Association convention.

Funding for this round came from existing angels, including BlueWater Angels, but also from RBV Capital, ExSight Capital and Santen Pharmaceutical.

Santen Pharmaceutical, based in Japan, has been developing therapies for vision disorders for more than a century – beginning with the basic eye drop. Since, it’s focused on glaucoma, and retinal and corneal disorders.

RetroSense received orphan status from the U.S. Food and Drug Administration for RST-001 to treat retinitis pigmentosa, as there are no approved drugs on the market to improve or restore vision in patients with this disorder.

It works by inserting a gene that improves light sensitivity to retinal nerve cells. It’s based on a gene that comes from algae, called channelrhodopsin-2, that helps pond scum to detect where the sun is shining to conduct photosynthesis. While the acuity is still up for testing, the gene’s been helpful in restoring functional vision in animal studies, the company says.

RetroSense is currently recruiting patients for the Phase 1/2 trial at the Retina Foundation of the Southwest.

Using gene therapies for vision restoration is the aim of several companies – including British startup NightstaRx, which raised $35 million earlier this week for a similar viral vector approach. And Editas Medicine plans to deploy the powerful CRISPR gene editing tool in human trials by 2017 to treat a congenital vision loss disorder. The reason this is likely a key area of focus for gene therapy is that gene therapies can be injected directly into the eye – showing local therapeutic benefit with little risk of impacting the rest of the body.

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