Testing Two New Therapies to Restore Sight in Advanced Retinitis Pigmentosa

Wednesday, May 31 2023 | 10 h 36 min | Vision Science

Kiora Pharmaceuticals has announced that they have dosed the first patient in a Phase 1 clinical trial for the photoswitch molecule KIO-301. This therapy is currently being tested in individuals with retinitis pigmentosa (RP). The molecule KIO-301 is designed to turn other retinal cells into light sensors and restore the ability of the eye to sense and interpret light signals. FBC supported development of related photoswitch molecules through a Restore Vision 20/20 Research Award to Dr. Richard Kramer (University of California Berkley).

In other clinical trial news, GenSight Biologics announced early results from their Phase I/II clinical trial, testing the optogenetic therapy GS030 for advanced RP. This potential treatment uses a gene therapy approach paired with special goggles. So far nine patients have received different doses of the therapy. At one year post gene therapy, the patients had no serious side effects. While the primary purpose of this trial is to test the safety of the therapy, some patients on the highest dose were able to locate and count objects, whereas before the therapy, they could barely sense light. The trial will be following patients to examine long-term safety before hopefully launching a trial to formally test if the treatment is effective.

Both treatments described above are gene-agnostic, meaning they could be effective for vision loss caused by many types of gene mutations. Current trials are only testing the therapies in patients with RP, but in the future if successful, it is hoped that these therapies could be used by individuals with other types of inherited retinal diseases.