Nanoscope® Therapeutics Inc., a TechFW client, has received an Orphan drug designation from the U.S. Food and Drug Administration (FDA) for gene therapy-based treatment of Stargardt disease, a form of inherited retinal degenerative disease caused by gene mutation and passed on to children.

Nanoscope is a clinical-stage biotechnology company determined to change lives through the ocular gene therapy. This is the second program based on its Multi-Characteristic Opsin Platform (MCO) to receive orphan designation.

"Currently, there are no approved therapies for Stargardt Disease" said Sulagna Bhattacharya, Chief Executive Officer of Nanoscope. "We are excited by the potential of ambient light activatable MCO photosensitization of retinal neurons for treating Stargardt disease in a gene agnostic manner."

Central vision loss because of poor functioning RPE cells – the pigmented layer of retina - and loss of photoreceptors in the macula is the hallmark of Stargardt disease. According to the Federation for Fighting Blindness, Stargardt disease is the most common form of inherited macular degeneration, affecting about 30,000 people in the U.S. There is an immense need to restore vision to these patients.

“Since the pathology of degenerated macula in Stargardt disease is similar to that of dry-AMD, we are very excited about the opportunity to learn from our Stargardt program and advance MCO-based, dry-AMD program," said Samarendra Mohanty, President and Chief Scientific officer of Nanoscope, which received an orphan designation in 2017 for MCO for treatment of Retinitis pigmentosa (RP).

The Orphan Drug Act encourages the development of medicines for rare diseases, and its benefits include tax credits and application fee waivers designed to offset clinical development costs, as well as eligibility for seven years of post-approval market exclusivity.

Nanoscope has received multiple SBIR awards, National Institutes of Health grants, and patents that focus on optical stimulation, gene delivery and imaging for neural activity monitoring.