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Nanoscope Therapeutics Receives Orphan Drug Designation for Gene Therapy of Blindness

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January 25, 2021

NANOSCOPE THERAPEUTICS today announced that it has received an Orphan drug designation from 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.

Central vision loss due to poor functioning RPE cells and loss of photoreceptors in the macula is the hallmark of Stargardt disease. According to Federation for Fighting Blindness, Stargardt disease is the most common form of inherited macular degeneration, affecting about 30,000 people in the U.S. The loss of vision is devastating to both children and adults, and significantly impacts their quality of life.  There is an immense need to restore vision in these patients.

“Currently, there are no approved therapies for Stargardt Disease” said Sulagna Bhattacharya, Chief Executive Officer of Nanoscope Therapeutics. “We are excited by the potential of ambient light activatable Multi-Characteristic Opsin (MCO) based photosensitization of retinal neurons for treating Stargardt disease in a gene agnostic manner.”

“vMCO-010 expands the portfolio of orphan drug designations obtained in multiple pipeline programs, expanding its therapeutic potential from Retinitis Pigmentosa (RP) with peripheral retinal degeneration to macular degeneration as in Stargardt disease”, said Al Guillem, Chairman of Nanoscope Therapeutics Board.

Sai Chavala, Chief Medical Officer of Nanoscope Therapeutics, commented “We look forward to developing our innovative optogenetic platform technologies for visually-challenged patients through continued interaction with the FDA.  The orphan designation will aid in accelerating our clinical development program.”

The Orphan Drug Act encourage the development of medicines for rare diseases, and 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.

“We are extremely excited and pleased to announce a second orphan drug designation for MCO based treatment for retinal diseases. 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 Therapeutics that received an orphan designation in 2017 for MCO for treatment of RP.

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