A new hope for restoring vision in retinitis pigmentosa patients


New Orleans, La. — Retinitis Pigmentosa (RP) is a group of inherited eye diseases (IRDs) that cause gradual vision loss. RP is a genetic disease with the onset of symptoms generally beginning in childhood. There’s no cure for RP, only visual aids and rehabilitation programs. This week at the Association for Research in Vision and Ophthalmology’s (ARVO) 2023 Annual Meeting, a study shared early information and results from a human clinical trial in blind and ultra-low vision patients receiving a first-of-its-kind molecule that has the potential for restoring vision.

This Phase 1b study tested the safety and effectiveness of KIO-301, a small molecule known to block certain ion channels in the eye to allow cell reactivation in animal models of degenerating retinas when exposed to light. It has also shown restored vision in animal models with RP. Trial participants with late-stage RP received a single dose of KIO-301 into the eyes and were monitored for 29 days. Initial subjects had positive outcomes and showed improvement in their functional vision.

Eric Daniels, MD, MBA, Chief Development Officer of the trial sponsor, Kiora Pharmaceuticals, commented that, “KIO-301 is a light-sensing small molecule that has the potential to restore vision to patients with inherited or age-related retinal degeneration. Early results demonstrated safety and profound improvements in light perception and functional vision evaluated by objective measures. This included object identification, peripheral vision and functional MRI scans, as well as patient-reported outcomes, such as surveys as to how vision is used in everyday activities.

  • Abstract title: An Intravitreal 'Photoswitch' Molecule (KIO-301) for Reanimation in Retinitis Pigmentosa: a first-in-human trial
  • Presentation start/end time: Thursday, April 27, 1:45 – 2:00pm CT
  • Location: 353-355
  • Presentation number: 5444


The Association for Research in Vision and Ophthalmology (ARVO) is the largest eye and vision research organization in the world. Members include approximately 10,000 eye and vision researchers from over 75 countries. ARVO advances research worldwide into understanding the visual system and preventing, treating and curing its disorders. Learn more at

Media contact:
Jenniffer Scherhaufer