In reporting on emerging therapies for LCA and other retinal diseases, my mantra has always been “just the facts.” If you’re old enough, you might remember those iconic words from the stoic detective Joe Friday on the TV show “Dragnet.” Bottom line, I prefer not to embellish my reporting with hyperbole or emotion. (But I don’t wear a trench coat.)
With that said, I can’t help but be genuinely excited by the news on LCA research that came from the 2026 Association for Research in Vision and Ophthalmology (ARVO) meeting in Denver, May 3-7. Nearly 11,000 research-focused professionals from 72 countries attended this year’s meeting. Not all the LCA reports were breaking news, but collectively, the presentations and posters by researchers from all over the globe were inspiring.
Here are some clinical development highlights from the meeting:
LCA13 (RDH12): InnoVec’s emerging RDH12 gene therapy performed encouragingly at 12 months in a Phase 1/2 clinical trial in China. Ten patients (three with LCA) received one of two doses. The vision of two patients with LCA went from only hand-motion perception to modest visual acuity. Innovec is working to launch a trial for the gene therapy in the U.S. Opus Genetics also plans to launch an RDH12 gene therapy clinical trial in the US by the end of 2026.
LCA5: Opus Genetics previously reported meaningful vision improvements in three adult and three pediatric patients (aged 16-17) in its Phase 1/2 LCA5 gene therapy clinical trial at the University of Pennsylvania. LCA5 is one of the rarest and most severe forms of LCA. Some patients saw objects for the first time after treatment. Others had meaningful improvements in visual acuity. Opus is now recruiting for its Phase 3 cohort and plans to dose younger pediatric patients.
LCA4 (AIPL1): LCA4 is extremely rare and severe. At an early age, many children have only light perception or hand-motion vision. Incredibly, researchers at Great Ormond Eye Hospital and Moorfields Eye Hospital restored meaningful vision in 11 patients (aged 4 or younger) with an LCA4 gene therapy developed by MeiraGTx in the U.K. under a Specials License. MeiraGTx has licensed the LCA4 gene therapy to Eli Lilly, which is pursuing regulatory approval for it.
LCA2 (GUCY2D): Atsena Therapeutics reported 36-month results for its 15-patient Phase 1/2 LCA2 gene therapy clinical trial. Patients receiving the highest dose maintained significant improvements in retinal sensitivity and other functional measures. Furthermore, there were no serious adverse events related to the therapy. The company plans to launch its global Phase 3 cohort in the second half of 2026.
LCA10 (CEP290, IVS26 mutation): Sepul Bio continues to recruit for its global Phase 3 HYPERION clinical trial for its RNA therapy, sepofarsen. At ARVO, a post-hoc, paired-eye analysis of the previous Phase 3 ILLUMINATE clinical trial for sepofarsen was presented. It showed that treated eyes performed better than untreated eyes in the same patient. Why is this important? In the original ILLUMINATE clinical trial, the treated eyes of patients were compared with untreated eyes in a different patient cohort (i.e., a control group). Ultimately, ILLUMINATE, completed in 2022, didn’t meet its primary endpoint. The good news: HYPERION uses a paired-eye design, which researchers believe can lead to a stronger efficacy signal and a better opportunity to meet the primary endpoint.
This is by no means an exhaustive list of ARVO research presentations on LCA. Please feel free to reach out to me at ben@hopeinfocus.org if you have any questions about these or other projects.