Clinical trial for light-controlled genes could open door for exciting therapies
RetroSense Therapeutics begin clinical-safety trials for optogenetic vision disorder treatment
Using light to control the activity of genes and neurons. This may sound like the stuff of science fiction, but optogenetics is in fact gearing up to enter mainstream medicine. Back in March, Ann Arbor startup RetroSense Therapeutics began the first clinical-safety trial of an optogenetic therapy, and many scientists are holding their breath for the results.
Restoring lost vision, managing pain and seizures, even treating brain disorders like Parkinson’s disease; if this trial is successful it could pave the way for some really exciting research developments.
Speaking to Nature, Washington University in St Louis pain researcher Robert Gereau said, “I think it will embolden people if there’s good news. It opens up a whole new range of possiblilities for how to treat neurological diseases.”
RetroSense are taking on retinitis pigmentosa, and disorder that destroys the photoreceptors in the eye and leaves the patient blind. Their therapy targets retinal ganglion cells, the messenger cells that normally transmit light sensitivity information to the brain but have no role in detecting light. By injecting these ganglion cells with a virus that genes for light-sensitive proteins called opsins, RetroSense hope that they can encourage these cells to fire when stimulated with blue light.
RetroSense are not the only players in the optogenetics space. In France GenSight Biologics are also going after retinitis pigmentosa using red light-sensitive opsins instead of blue. And in Menlo Park, California, Circuit Therapeutics are exploring a wide range of therapeutic applications, including the management of pain.