On Oct. 5, Karl Deisseroth, MD, PhD, and two other scientists won the 2026 Nobel Prize in Physiology or Medicine.
The recognition was for the discovery and impact of optogenetics. Optogenetics introduces genes for light-sensitive proteins into cells and then uses light to activate or deactivate cells in living tissue.
The California Institute for Regenerative Medicine (CIRM) provided early support for Deisseroth’s pioneering work. That work helped establish a transformative approach to controlling cell behavior. It opened up new possibilities for understanding behavior and neurobiology, and ultimately for developing new therapies.
In 2008, CIRM supported Deisseroth with a $2.4 million New Faculty Award. That award helped him establish his Stanford Medicine lab and improve optogenetic tools used in stem cell research. He is now a Stanford University professor in the School of Medicine and the School of Engineering,
Optogenetics
Now, that foundational research is applied across medical fields in thousands of labs around the world.
“Optogenetics is an engine for discovery; it’s for probing complex systems at the fundamental level of cells,” Deisseroth told Stanford Medicine in a recent press release. “Once you know the cells that are important in a symptom or in correcting a symptom, then you can design any method you like to target their activity.”
The New Faculty Awards were intended to provide salary and research support for up to five years, creating a stable environment for these new faculty members to build innovative and robust stem cell research programs in the state of California.
During the early discovery phase, CIRM funded Deisseroth, validating its ability to uncover important aspects of human biology. Since that early award, the field has broadened beyond basic human biology to testing optogenetics as a possible therapy for eye and heart diseases.
“With the CIRM New Faculty Award support, and that of other funders, Deisseroth established his new lab at Stanford University and rapidly developed new optogenetic tools,” said Uta Grieshammer, the science officer on Deisseroth’s 2008 New Faculty Award. “Today, those tools are applied to develop treatments for vision loss in human patients, a testament to how basic discovery research can advance to clinical application with CIRM support.”
Some examples from the CIRM portfolio that target major vision loss with optogenetics include:
Ray Therapeutics
$6.6 million for Stargardt disease (a type of juvenile macular degeneration)
$8 million (announced last month) for Stargardt disease
$8 million for retinitis pigmentosa
$4 million for age-related macular degeneration (AMD)
$4 million for retinitis pigmentosa and other inherited retinal diseases
Adverum Biotechnologies
$1 million for developing melanopsin-based gene therapy for vision restoration