Congratulations to Professor Karl Deisseroth (The Howard Hughes Medical Institute and Stanford University), Professor Peter Hegemann (Humboldt University of Berlin) and Professor Georg Nagel (University of Würzburg), who were jointly awarded the 2026 Nobel Prize in Physiology or Medicine for their discoveries concerning light-gated ion channels and their pioneering contributions to the field of optogenetics.
From single-celled algae to brain switches
Their work began with Professor Hegemann’s curiosity about how the single-celled alga Chlamydomonas is able to swim towards a light source and, remarkably, why it responds to light more than twenty times faster than the human eye. Professor Hegemann proposed that the alga uses a single protein that both captures light and functions as an ion channel. In the early 2000s, Professor Hegemann and his colleague Professor Nagel proved the hypothesis correct. Professor Nagel expressed the algal genes in frog eggs and showed that the proteins they encode, named channelrhodopsin-1 and channelrhodopsin-2, are ion channels that open in response to light, generating an electrical signal.
Channelrhodopsin-2 was of interest to Professor Deisseroth, a psychiatrist looking for a molecular tool to study the function of nerve cells in the brain, in order to understand diseases such as depression, autism and schizophrenia. In 2005, Professor Deisseroth’s group successfully expressed the channelrhodopsin-2 gene in rat nerve cells, demonstrating that the cells became sensitive to blue light and could propagate electrical signals to other nerve cells. In 2007, Professor Deisseroth’s team expressed channelrhodopsin-2 in mice and showed that whisker movement could be controlled by delivering light through an optical fibre to switch on a selected population of neurons in the brain. Further research refined the technology, enabling the mapping of neural pathways and the identification of specific cell populations responsible for different brain functions.
Named optogenetics in 2006, the technology has also become an important tool for studying how the nervous system controls tissues outside the brain.
Beyond neuroscience and into medicine
Optogenetics is now used to identify precisely which neuronal circuits are responsible for disease symptoms and shows potential for identifying novel drug targets across a broad spectrum of psychiatric and neurological disorders, including depression, anxiety, schizophrenia, Alzheimer’s disease and Parkinson’s disease.
Commercial interest in optogenetics is growing and the first approved optogenetic therapy could arrive as early as next year. Last month, the biotechnology company Nanoscope Therapeutics announced that the US FDA had accepted its Biologics License Application (BLA) for Mogenry® (sonpiretigene isteparvovec), following favourable results in Phase 2b/3 development. Mogenry® is a gene therapy for the treatment of retinitis pigmentosa that delivers a gene encoding a synthetic light-sensitive opsin to retinal bipolar cells, making them responsive to light. An FDA decision is expected in the first half of 2027. Nanoscope licensed CatCh, one of Mogenry’s three light-sensitive components which is an engineered variant of channelrhodopsin-2, from Max Planck Innovation (the technology transfer arm of the Max Planck Society). CatCh was developed by Professor Ernst Bamberg and colleagues at the Max Planck Institute of Biophysics in Frankfurt, the same institute where Professor Nagel, Professor Bamberg’s former PhD student, made his prize-winning discoveries.
Two other notable companies developing therapies for retinitis pigmentosa are Ray Therapeutics, which has developed its own optogenetic gene therapy candidate, RTX-015, currently undergoing Phase 2/3 development, and GenSight Biologics, which combines gene therapy with wearable goggle technology. As commercial interest grows, we can expect next-generation light-sensitive proteins and platforms, and expansion into related eye disorders.
Beyond eye disorders, light-sensitive proteins such as channelrhodopsin are being explored in preclinical research for applications including optical cochlear implants for hearing loss, making heart tissue light-sensitive to control arrhythmias, and light-triggered suppression of epileptic seizures.
The Nobel Prize winner Professor Deisseroth also co-founded the biopharmaceutical company MapLight Therapeutics, which uses an optogenetics-based drug discovery platform and has lead candidates in Phase 2 development. Optogenetics-guided drug discovery could have the broadest commercial reach of the technology, because the resulting drugs avoid the challenges associated with gene therapy, do not require delivery of light to tissues, and the approach has the potential to generate a much larger number of therapeutic candidates.
It will also be interesting to see how optogenetics combines with other technologies in the future, in particular, pairing neural pathway mapping with single-cell and spatial gene-expression data, as MapLight Therapeutics has done, and AI-driven target selection which has the potential to speed up the pathway from identifying a neural circuit associated with disease to identifying drug candidates that act on it.
Author: Oliver Lam
Oliver provides tailored strategic and commercial advice on a diverse range of technologies, including pharmaceuticals, immunotherapies, gene therapies, RNA therapeutics and diagnostic biomarkers. He has considerable experience in mRNA technology, particularly vaccine formulation, having supported a large international litigation relating to COVID-19 vaccines.
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