Hope in Focus Updates from the Association for Research in Vision and Ophthalmology Annual Meeting
Hope in Focus Director of Outreach and Development Courtney Coates attended the ARVO Annual Meeting on behalf of the organization to learn more about current LCA research to raise awareness about Hope in Focus, and network with young LCA researchers. Hearing another year filled with updates on LCA programs and inherited retinal diseases was exciting…
Some specific updates relevant to the LCA community are below.
Dr. Allen Ho from Wills Eye Hospital and Mid Atlantic Retina presented a paper on the one-year results of Nanoscope Therapeutic’s gene-agnostic optogenetics treatment for retinitis pigmentosa. Patients in the Phase 2b/3 trial showed improvements on the Freiburg Visual Acuity Test and with the multi-luminance mobility test. Nanoscope Therapeutics plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024. Nanoscope Therapeutics is currently running a Phase 1/2a trial with the same asset for Stargardt’s disease and plans to add LCA as an indication in the future.
Dr. Tomas Aleman presented on the first three patients dosed in the clinical trial for LCA5 run by Opus Genetics. The patients were given a low dose of a traditional gene therapy delivered via AAV. One of the participants went from only counting fingers to being able to read a letter on the eye chart. The other two participants did not see a significant change in visual acuity. The youngest participant had significant improvement in the virtual reality mobility course, which is used to measure a meaningful gain in functional vision. The trial will enroll three more participants for the mid-dose in their next cohort.
Dr. Byron Lam presented on “OCU400, Ocugen’s Nuclear Hormone Receptor-Based Gene Modifier Therapy for Retinitis Pigmentosa Associated with NR2E3 and RHO Mutations in their Phase 1 / 2 Clinical Trial.” Data from the study supported a good safety profile with no serious adverse events and early signs of efficacy. A later cohort added four patients with LCA CEP290 but data for that cohort is not yet available. Ocugen announced that it will be moving forward with a Phase 3 trial for OCU400.
Dr. Alvin Luk presented on their Phase 1 study in China for HG004, a gene therapy for mutations in the RPE65 gene. The therapy uses AAV9 for delivery and uses a smaller volume of injection made into multiple blebs. So far, there have been no adverse events and no signs of chorioretinal atrophy. Early efficacy results showed improvements in the Multi Luminance Mobility Testing course, and visual acuity improvements were similar to the gains achieved with LUXTURNAⓇ.
