RetroSense Closer to Clinical Trials After Orphan Drug Designation

Recent action by the FDA has Ann Arbor, MI-based RetroSense Therapeutics looking to possible clinical trials next year for a gene therapy that could help people see better.

The key ruling came last week when regulators granted orphan drug status to RST-001, which RetroSense is developing to restore vision for people affected by retinitis pigmentosa.

Orphan status is granted to kickstart the development of treatments for diseases that affect fewer than 200,000 people in the Unites States. The designation comes with increased market exclusivity, reduced regulatory fees, and tax credits.

The active ingredient in RST-001 is a photosensitivity gene called Channelrhodopsin-2 (ChR2), which is found in blue-green algae. RetroSense has demonstrated in tests involving mice and monkeys that ChR2 can be administered into a degenerated retina to restore vision, RetroSense CEO Sean Ainsworth says. The technology was originally developed at at Massachusetts General Hospital and at Wayne State University by anatomy and cell biology professor Zhuo-Hua Pan.

“The rods and cones die out, and that’s what normally senses light,” Ainsworth says. “RST-001 delivers the ChR2 gene to the eye, and by targeting the retinal neurons that survive, we can confer light sensitivity to those neurons.”

With the new orphan drug status, RetroSense hopes to initiate the clinical studies process sometime in 2015. Ainsworth says he’s particularly excited about the prospect of being able to see signs of RST-001’s efficacy fairly early in the studies. “We’re proposing to restore vision instead of slowing the process of disease,” he explains.

That’s not the only exciting news for RetroSense: Ainsworth says the company’s Series A round, which is set to close next month, is already “pretty substantially oversubscribed.” In June, RetroSense announced it had already raised $2.4 million in VC capital, led by the San Diego-based firm Nerveda.

“We originally raised a few million, but we’ve gone way past that,” he says. “We initially got things started with Blue Water Angels, and they connected us to investors on the West Coast.”

For now, Ainsworth says RetroSense is moving toward clinical trials as a lean operation. The number of employees hasn’t grown much by design, he adds, in order to keep the company’s expenses down.

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