Phase 1/2 trial update for Leber congenital amaurosis gene therapy in pediatric patients

Opus Genetics provided an update on its Phase 1/2 clinical trial evaluating OPGx-LCA5, an investigational gene augmentation therapy, as a treatment for Leber congenital amaurosis type 5 in pediatric patients. The three patients who were between 16–17 years old had severe baseline vision impairment and received one subretinal injection of OPGx-LCA5. According to the company’s press release, all 3 showed improvements in visual acuity (0.3 logMAR improvement, which is greater than that observed in the adult cohort), greater than 1 log unit improvement in cone sensitivity to red and blue light (suggesting recovery in retinal sensitivity), and identification of more objects through 3 months compared to baseline. The company stated that the therapy was well tolerated with no serious ocular adverse events or dose-limiting toxicities. To date, three adults and three pediatric patients received OPGx-LCA5. A meeting with the FDA to discuss the results and next steps for the program is expected in the fourth quarter of this year.

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Primary endpoint not met in Phase 2b trial investigating persistent corneal epithelial defect treatment

KALA BIO announced that its Phase 2b trial investigating KPI-012 for treatment of persistent corneal epithelial defect did not meet its primary endpoint. According to the company’s press release, it also did not achieve statistical significance for its secondary endpoints. It was well tolerated with no treatment-related serious adverse events. The company has stopped clinical development of KPI-012 and its mesenchymal stem cell secretome platform.

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Study: treatment for uveal melanoma could improve survival rate

A study, led by researchers at the University of California, Los Angeles, published in the journal Cancers, found that a treatment technique for uveal melanoma could reduce metastasis risk and improve survival rates. According to the paper, between 30–35% of patients who have uveal melanoma experience metastasis and die within 5 years. In this study, a single ocular oncologist treated patients with uveal melanoma (n=37). Most (n=27) were treated with plaque radiation (brachytherapy) and vitrectomy with silicone oil replacement, which is used to reduce the effect of radiation on vision. Seven patients had brachytherapy without vitrectomy/silicone oil, and 3 were treated with enucleation. These patients were followed up with for 4.0±3.7 years. According to the paper, 16.2% (6 patients) developed metastasis and one patient died due to metastatic disease. The authors, who evaluated prognostic markers associated with increased metastasis risk (tumor dimensions and monosomy 3 and/or Class 2 gene expression of the tumor), found “an unexpectedly low rate of 5-year metastasis” … and reported that “AJCC stage correlated poorly with metastasis or survival.” The university’s news article about the study noted “the potential benefits of combining vision-preserving surgical techniques with targeted radiation therapy, [but] the researchers caution that more research is needed to determine whether the surgical technique, radiation delivery, or a combination of factors contributed to the improved outcomes and understand exactly why outcomes were so favorable.”

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Company news

• Harrow announced its intent to acquire Melt Pharmaceuticals, the latter of which is developing MELT-300, an investigational sublingual formulation of midazolam (3 mg) and ketamine (50 mg) for sedation and analgesia in hospital, outpatient, and in-office settings.
• DefEYE launched as an independent company, previously operating as Verséa Ophthalmics, a subsidiary of Verséa Health. DefEYE has exclusive rights to a portfolio of decellularized biologics, including BIOVANCE products.

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ASCRS news and events

• ASCRS Live!: This educational dinner series is heading to several cities across the U.S. in 2025. Registration is open for the next event in Orlando on October 16.
• 2026 ASCRS Annual Meeting: Hotel room blocks are open for the 2026 ASCRS Annual Meeting, taking place in Washington, D.C., April 10–13, 2026. The call for case video submissions is going on now—learn more. Registration will open later this month.

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Research highlights

• The visual outcomes of eyes treated with ocular wavefront-guided LASIK (planned with the iDesign Advanced WaveScan Studio System, Johnson & Johnson Vision) or topography-guided LASIK (planned with the Contoura Vision and Wave Light Topolyzer Vario, Alcon) were evaluated in a prospective, randomized, contralateral eye-controlled trial. According to the paper published in the Journal of Cataract & Refractive Surgery, the study included 100 eyes of 50 patients with myopia or myopic astigmatism who were treated between January 2023 and April 2024; of these, 96 eyes of 48 patients completed 12 month follow-up. At 12 months postop, there were no statistically significant differences between the two groups when it came to spherical equivalent, higher order aberrations, proportion of eyes that had 20/20 or greater UDVA, or proportion of eyes that had improved 5% and 25% low-contrast visual acuity. A greater percentage of eyes that achieved UDVA of 20/12.5 or greater was observed in the wavefront-guided group compared to the topography-guided group, which was significant, according to the authors.
• A systematic review and meta-analysis published in the American Journal of Ophthalmology investigated the safety and efficacy of crosslinking for patients with keratoconus who have a corneal thickness of less than 400 μm. Twenty-nine studies were included in the analysis (most being retrospective case series with “substantial potential for bias) between 2011 and 2024 for a total of 470 eyes. The authors noted “substantial heterogeneity” between the studies when it came to the patients, the crosslinking approach, and outcomes. The authors reported that flattening of the maximum corneal curvature and improvement in corrected distance visual acuity were observed in these studies, compared to baseline, and no study reported serious complications. This, they concluded, suggests that crosslinking could be “suitable for patients with stromal thicknesses of <400 μm, providing adequate modifications have been applied.” They advocated for more prospective comparative studies.

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Product news

• VIZZ (aceclidine ophthalmic solution, Lenz Therapeutics), 1.44%, is now available, with commercial shipments to consumers through the company’s ePharmacy partner beginning in the fourth quarter of 2025.
• OCuSOFT issued a clarification on its OCuSOFT Retaine MGD product, noting that it discontinued its previous formulation (branded as Retaine MGD) in 2024 and replaced it with an “advanced formulation” that’s branded and available as Retaine MGD Advanced.
• Neurotech Pharmaceuticals was assigned a permanent J-Code (J3403) for ENCELTO, effective October 1, to treat idiopathic Macular Telangiectasia Type 2.
• Lumibird Medical launched ICL sizing assistance, compatible with ICL Guru (REVAI), on its ABSolu ultrasound platform.

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