Small molecules, in contrast to gene therapies and other biological based therapeutics, are the most common type of drugs approved throughout the world. This translates to a shorter development timeline when compared to these more complex approaches (e.g. gene therapy).
KIO-301 specifically enters key downstream retinal cells (those no longer connected to viable rods and cones) and turns them into light sensing cells, capable of signaling the brain as to the presence or absence of light.
The ability to detect the presence or absence of light is the foundation of vision, and unfortunately, is lost in a number of key degenerative diseases of the eye. The normal human retina has about 120 million rods (black & white, night vision, movement) and 6 million cones (color). In certain retinal diseases, photoreceptors (rods and cones) die (rods first, then cones), rendering them unable to activate downstream Bipolar and Retinal Ganglion Cells (“RGCs”). Photoreceptors normally sense light and convert light energy into electrical energy to pass the signal to special transmitting cells. Bipolar cells and RGCs relay the signal from the rods and cones to the brain, where the image is “processed”.
In patients with Retinitis Pigmentosa (RP), photoreceptors are no longer viable and therefore their downstream “signal relaying” cells (RGCs) are not capable of being activated. KIO-301 preferentially enters these RGCs and turns them into light sensing cells. KIO-301 achieves this by lodging inside specific voltage-gated ion channels. In presence of light, physically blocks ion outflow, causing cell depolarization and hence signaling the brain. When light is turned off, KIO-301 reverts to its lower energy state which causes the cell to repolarize and “stop” signaling the brain.