Nanoscope’s Optogentic Gene Therapy

Nanoscope’s Therapeutics announced in June the results of an Optogenetic Gene Therapy trial to restore meaningful vision in 11 Patients blinded by Retinitis Pigmentosa.

In most cases of retinal photoreceptor cell damage, the neural layer that sends messages to the brain remains relatively intact. These cells do not react to light but research efforts over the past few years are rapidly changing this to make these cells, and particularly the ganglion cells, light responsive.

Nanoscope Therapeutics Inc is developing a gene therapy to deliver a light sensitive molecule [MCO] to the neural cells. They recently announced vision improvements for patients with advanced Retinitis Pigmentosa [RP] in a phase 1/2a clinical trial. The improvements persisted 52 weeks after a single intra vitreal injection of Multi-Characteristic Opsin. This is the first reported clinically meaningful functional improvement by optogenetic therapy.

This vision restoration was observed in normal ambient light using optogenetic gene monotherapy without the need of stimulating retinal implants or external devices such as goggles.

The beauty of this approach is that it would potentially work in patients affected by multiple genetic defect(s) in a mutation-independent manner. Significant improvement in multiple mobility tasks and quality of life measures was reported. A phase 2b, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial is expected to be launched later this year.