vision in retinal degeneration
innovative optogenetic gene therapy
ARCTOS medical is developing ARC004 for the treatment of blindness associated with retinal degenerative diseases.
ARC004 belongs to the class of optogenetic gene therapies. Such therapies selectively introduce genes encoding light-sensitive proteins into surviving retinal cells to act as “replacement light sensors”.
The therapeutic principle is Opto-mGluR6*, a chimeric all-retinal G-protein-coupled-receptor (GPCR).
This chimera is consisting of the intracellular domains of the ON-bipolar cell—specific metabotropic glutamate receptor mGluR6, and the light-sensing domains of melanopsin.
For delivery of Opto-mGluR6 Arctos Medical is using an Adeno-virus variant targeting Bipolar cells in the inner Retina and a cell-specific promoter.
*Restoring the ON Switch in Blind Retinas: Opto-mGluR6, a Next-Generation, Cell-Tailored Optogenetic Tool, M van Wyk,, J Pielecka-Fortuna, S Löwel and S Kleinlogel; PLoS Biol. 2015 May; 13