Nov 7, 2023
Brian Strem, President and CEO of Kiora Pharmaceuticals, is developing a small molecule drug to treat orphan retinal diseases. Their drug KIO-301 restores vision in patients with inherited retinal disease. By giving retinal ganglion cells the ability to sense light, signals can be sent to the brain to effectively see using cells that are not normally light-sensitive. These molecular photoswitches can change shape based on the presence or absence of light and allow a patient to see the direction and movement of light.
Brian explains, "It is dependent on the specific mutation. So, we know that there are certain gene mutations that a child will literally be born with immediate signs and symptoms of that disease. Whether that is immediately being blind, so being born blind, versus some of the other ones where you don't tend to get the onset of disease symptoms until the second, or third decade of life when you have difficulty seeing in the dark. Then all of a sudden, that progresses to losing peripheral vision and eventually central vision. So, it is pretty specific to the mutation as to the onset, the timing of onset, and the progression rate."
"KIO-301 is based around what chemists would call an Azobenzene. Azobenzenes are notorious for changing their shape based on different external stimuli. And in the case of our molecule, that stimulus is light within the visible spectrum. So, getting more towards, well, what does that mean, and how could we leverage that interesting chemistry? Patients who have these IRDs and specifically, we've now just wrapped up a phase 1 trial, which I can get into in a minute, for a disease called retinitis pigmentosa. In patients with RP, we know that their photoreceptors, or their rods and cones, die. That's part of that mutation that these patients are born with."
"Yet there are other cells within the retina that still are the ones that connect to the brain, and those are called the retinal ganglion cells. We know that those cells stay alive for a long time, although they undergo some changes and modifications. And what our molecule can do, KIO-301, is it goes specifically into those retinal ganglion cells that do not have living photoreceptors upstream anymore because they're dead. And what the molecule does is it gives that retinal ganglion cell the ability to sense light and to signal the brain that light is present. And then, when you take the light away, it's able to basically stop that signaling. So, in the sense that it's turning a cell that's not normally light sensitive into a light switch."
#KioraPharmaceuticals #VisionAwarenessMonth #InheritedRetinalDisease #RareDisease #OrphanRetinalDiseases
