Pharmaceutical Technology

Scientists claim Nobel Prize for approach behind light-activated gene therapy

The Nobel Prize committee says that optogenetics will lay the foundations for a new era in neuroscience, while pharma explores the method’s vision-restoring potential.

A trio of German and American scientists have won this year’s Nobel Prize in Physiology or Medicine for their discovery of a new brain activity-mapping and nerve signal-controlling method – giving credence to an approach pharma and biotech are currently exploring to treat several ophthalmic conditions.

Through this landmark win, Karl Diesseroth of Stanford University, Peter Hegemann of Berlin’s Humboldt University and Georg Nagel of the University of Würzburg will split a prize pot of SKr 12m ($1.19m), which the Nobel committee is awarding to the scientists for their work on discovering and developing the field of optogenetics.

At the core of the optogenetic approach is channelrhodopsin, an ion channel protein found in single-celled algae that opens upon exposure to blue light. After channelrhodopsin’s initial discovery in the early 2000s, the three Nobel Prize winners realised that the protein could be used to map the activity of nerve cells and how they shape the creation of memories, feelings and behaviours in the living brain. This gave rise to optogenetics as we know it today, which is being used to guide research into neurological and psychiatric disorders, among other things.

According to Per Svenningsson, chair of the Nobel Committee for Physiology or Medicine, optogenetics provides scientists with “opportunities for mapping the brain that we could once only dream of”. However, drugmakers are now betting that the approach will prove useful in the field of ophthalmology.

This has led several biotech and pharma companies to expllore the promise of optogenetics in a clinical setting, with several drugs currently in development as vision-restoring treatments for patients with certain degenerative eye conditions.

The most advanced medicine harnessing this technique is Nanoscope Therapeutics’ optogenetic gene-agnostic therapy, Mogenry (sonpiretigene isteparvovec), which is designed to restore vision in patients with severe retinal degeneration. Nanoscope created the one-time medicine to introduce a light-sensitive opsin protein to intact visual signal-transmitting cells in the eye, allowing them to better respond to light and improve vision despite photoreceptor death.

Thus far, the drug has already shown late-stage promise in both retinitis pigmentosa (RP) and Stargardt disease, with US regulators having accepted the biologics license application (BLA) for MCO-010 in RP back in September.

However, Nanoscope is not the only company looking to bring an optogenetic drug to patients, as companies like GenSight Biologics and Ray Therapeutics are also evaluating their gene therapies, GS030 and RTx-01, in RP clinical trials.

Currently, Roche-owned Spark Therapeutics and Novartis’ Luxturna (voretigene neparvovec) is the only vision-restoring therapy approved by the US Food and Drug Administration (FDA) for the treatment of RP. Analysts at GlobalData, the parent company of Pharmaceutical Technology, estimate that Luxturna’s sales will approach the $400m mark in 2032.