Nanoscope Therapeutics Inc., a Texas-based biotechnology company involved in developing and commercialising novel gene-agnostic therapies for patients suffering vision loss from retinal degenerative diseases, announced on Thursday the publication of positive results from its STARLIGHT clinical trial in eClinical Medicine, part of The Lancet Discovery Science.

The company says that STARLIGHT is the first and only clinical trial to demonstrate vision improvements in patients with Stargardt disease (SD).

STARLIGHT is a Phase 2, open-label study designed to assess the safety and efficacy of a single intravitreal injection of gene-agnostic MCO-010 therapy in six participants with severe vision loss due to SD.

Stargardt disease, also known as juvenile macular dystrophy, is a major cause of vision loss in children, affecting more than 40,000 people in the US and leaving over 20,000 legally blind. In patients with SD, the light-sensing photoreceptors in the macula degrade, leading to loss of central vision. Currently, there are no approved treatments for the disease.

"While this was an open-label study, this is one of the first studies to demonstrate vision improvement in Stargardt disease patients. This is a remarkable finding as these patients typically experience irreversible central vision loss over their lifetimes," added Byron Lam, MD, professor of Ophthalmology at Bascom Palmer Eye Institute, lead author and principal investigator of the STARLIGHT study.

The published manuscript, titled 'Safety and Efficacy of MCO-010 Optogenetic Therapy in Patients with Stargardt Disease in USA (STARLIGHT): an Open-label Multi-Center Ph2 Trial', is now available online and will appear in an upcoming print issue of The Lancet.