KIO-301 | Inherited Retinal Disease

KIO-301 is a new type of therapy under development for inherited retinal diseases that acts as a “molecular photoswitch.”

Diseases including retinitis pigmentosa (RP) cause the eye’s light-sensing cells (rods and cones) to degenerate, leading to vision loss. KIO-301 targets surviving cells in the retina (the Retinal Ganglion Cells or RGCs), which connect the eye to the brain. The molecule enters these cells and, when activated by light, allows them to send signals to the brain, aiming to restore visual function.

Thus, like a light switch, KIO-301 has “on” and “off” positions controlled by the presence or absence of light.

A clinical trial (ABACUS-2) is underway to evaluate KIO-301 in patients with RP who have profound vision loss. Patients with any of the 100+ genetic mutations associated with retinitis pigmentosa are potentially eligible.

Healthy Vision

Photoreceptors (rods and cones) process light and relay a signal downstream to retinal ganglion cells (RGCs). RGCs transmit these signals to the visual cortex to create the perception of sight.

Damage from Retinitis Pigmentosa

RP causes progressive degeneration of photoreceptors, leading to vision loss and, often, blindness. Importantly, because RGCs remain viable, they’re an ideal drug target.

Without Light

KIO-301 selectively targets ions on the surface of RGCs, where it can enter these cells. In the absence of light, KIO-301 remains in its linear “off” position.

With Light

When exposed to light, KIO-301 flips into its “on” position. This disrupts the flow of ions, which activates RGCs to process and relay light signals to the visual cortex.

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