Knowledge of a rare genetic disorder combined with gene editing tech could lead to a non-addictive treatment for pain.
Rare genetic diseases involving mutation of the SCN9A gene have helped researchers find a new target for pain treatments. SCN9A encodes Nav1.7, a sodium ion channel present in nerves that plays a role in transmitting pain signals to the brain. Some mutations of SCN9A lead to feelings of excessive pain, while others result in the elimination of all pain sensation.
No one has yet been successful at mimicking the pain-free effect using conventional drugs to block Nav1.7, although many companies have tried. A few companies are investigating potential gene therapies, including Voyager Therapeutics and Coda Biotherapeutics.
Startup Navega Therapeutics is the first to apply a variant of CRISPR (and ZFs) that acts as an epigenetic modulator to treat pain. The company was formed to commercialize technology developed at the University of California, San Diego by Ana Moreno, who is now its CEO.
The use of CRISPR (clustered regularly interspaced short palindromic repeats) gene-editing technology for the development of human gene therapies is only in the earliest stages, with the first development therapies recently undergoing human clinical trials.
Navega will (ideally) inject the cerebral spinal fluid with viral particles carrying a modified version of CRISPR designed to interrupt pain signals and temporarily block a key molecule in pain-transmitting neurons in the spinal cord. It has demonstrated the effectiveness of the treatment in mice. Reportedly, pain sensations are reduced, but not fully eliminated.
So far, CRISPR is only being investigated for use in gene therapies to treat rare diseases. Its use in pain treatment would lead to much wider use.