Nanoscope Announces topline results from randomised phase 2B trial of MCO-010 for retinitis pigmentosa

Nanoscope Therapeutics has announced positive results from their two year randomised controlled phase 2b optogenetic therapy trial for late-stage retinitis pigmentosa (RP).

Optogenetics is not dependent on genetic diagnosis but is only appropriate for advanced stages of sight loss. It works by using gene therapy techniques to provide cells at the back of the eye with the genetic instructions to build a light sensitive protein. These cells do not normally sense light, but are unaffected by RP so could potentially take over from degenerated photoreceptors.


The trial treatment, called MCO-010, is a gene-agnostic therapy meaning that this treatment could be appropriate for a large proportion of our community. However, it is aimed at those who have already experienced significant sight loss: Retina UK understands that trial participants’ baseline vision was ability to count fingers or less (e.g. hand movements or light perception only). The positive effects seen in the trial would not necessarily be replicated in those with better vision.

MCO-010 led to improved visual acuity and visual function in participants after a one year follow up.  After a 76 week follow up, visual acuity improved for patients in the high-dose group but there was no difference in visual acuity between the low-dose and control group at this point MCO-010  was associated with no major adverse effects.

Nanoscope have said they are possibly going to apply to the FDA later this year, however it is extremely unusual for regulatory authorities to license a product on such little data without a phase 3 trial. Meanwhile, other non-gene specific treatments for late-stage sight loss are being investigated by companies such as GenSight, Bionic Sight and Kiora Pharmaceuticals, each with slightly different approaches and consequent advantages and disadvantages.

Support our work

Support us to help more people on their sight loss journey.

Donate now