Phase 2 trial is the ‘first and only’ to show vision improvement for Stargardt disease

Nanoscope Therapeutics announced publication of positive results from its Phase 2 trial online in the journal eClinical Medicine ahead of print in The Lancet. The trial, according to the company, is the “first and only clinical trial to demonstrate vision improvements in patients with Stargardt disease.” The open-label study assessed safety and efficacy of MCO-010 (sonpiretigene isteparvovec) in 6 participants with severe vision loss due to the disease. In all patients there was a mean improvement of +5.5 and +9.0 ETDRS letters without and with a wearable low-vision aid, respectively, the company reported. For patients who had atrophy confined to the macula, there was a mean BCVA improvement at week 48 of +12.0 and +32.0 ETDRS letters, without and with a wearable low-vision aid, respectively. There were no serious adverse events during the 48-week study period. The company plans to begin a Phase 3 trial by the end of this year with topline results expected in 2027. MCO-010 is described as “a one-time, in-office intravitreal gene-agnostic therapy designed to restore vision in patients with advanced retinal degeneration.”

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IND accepted for retinal disease gene therapy and Phase 1/2 trial to begin

Opus Genetics announced FDA acceptance of its Investigational New Drug Application (IND) for OPGx-BEST1, which it described as a gene therapy for treatment of bestrophin-1-related inherited retinal diseases. The company described Best disease as vitelliform macular dystrophy caused by an inherited mutation in the BEST1 gene. The company stated that it will begin a Phase 1/2 trial in the second half of this year. The study will be multicenter and open label to assess safety, tolerability, and preliminary effect of one subretinal injection of the gene therapy. Functional and anatomical endpoints (changes in visual function and retinal structure) will also be assessed.

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First patients dosed in trial evaluating retinitis pigmentosa treatment

jCyte announced patient enrollment and first patient dosing in its Phase 2 trial evaluating safety and efficacy of jCell (famzeretcel) for treatment of retinitis pigmentosa. According to the company’s press release, the dose in this trial (an 8.8 million cell injection) is about 50% higher than the highest dose used in prior trials. jCell is not dependent on genetic subtype. The trial will enroll up to 60 patients with any genetic subtype of retinitis pigmentosa, randomizing participants to either receive the jCell therapy or sham treatment. Safety and vision changes will be assessed over 6 months. An extension study is planned following the conclusion of this trial for longer-term monitoring and the possibility for patients in the sham group to receive jCell treatment.

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LASIK alternative being researched

Presented at the meeting of the American Chemical Society (ACS), Michael Hill, PhD, and Brian Wong, MD, described their research involving a possible LASIK alternative called electromechanical reshaping. Currently in its early stages of research and development, they have performed the procedure ex vivo on 12 rabbit eyes, 10 of which served as a myopia model. According to the news release from the ACS, the procedure involves “specialized, platinum ‘contact lenses’ that [provide] a template for the corrected shape of the cornea,” which were then placed over the ex vivo eye in a saline solution. The platinum lens acts as an electrode “to generate the precise pH change” in the cornea when a small electrical pulse was applied. Shortly thereafter, the corneal curvature conformed to the lens shape. According to the release, the cells survived treatment. The research will be conducted in vivo on rabbit eyes and will include evaluating if hyperopic and astigmatic corrections are possible.

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Partnership news

• Boehringer Ingelheim and Palatin Technologies announced their collaboration and licensing agreement to develop melanocortin receptor agonists for treatment of retinal diseases, including diabetic retinopathy.

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ASCRS news and events

• Business of Refractive Cataract Surgery (BRiCS) Summit: This course, taking place October 3–5, in Chicago, Illinois, along with its extensive, take-home Resource Library, will help practices gain the navigational tools needed to increase premium IOL adoption and lead patients effectively on their refractive surgery journey. Learn more.
• Business of Interventional Glaucoma: This program seeks to provide ophthalmology professionals with the tools and strategies needed to effectively manage and grow interventional glaucoma services. The next program will take place on Thursday, October 16 from 9:00 a.m.–4:00 p.m. at the Rosen Centre in Orlando, Florida. Learn more.
• 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 Philadelphia on September 11.
• 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 submissions is also ongoing until September 1. Submit an abstract and get involved in the 2026 ASCRS Annual Meeting.

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Research highlights

• A study evaluating the safety and efficacy of scleral allograft-reinforced cyclodialysis in an interventional single-center case series with 24 months of follow-up was published in the journal Ophthalmology Science. The study included 31 eyes with primary open-angle glaucoma and cataracts that had the bio-interventional cyclodialysis procedure along with phacoemulsification cataract surgery. The primary outcome (a 20% or greater reduction in IOP) was achieved in 74% of eyes; the mean IOP reduction from baseline was 34%. At 12 months postop, mean IOP was 12.62±2.63 mm Hg on 0.55±0.52 IOP-lowering medications, a reduction from 21.9±4.92 mm Hg on 1.22±1.29 IOP-lowering medications at baseline.
• Epithelial and stromal changes in patients experiencing post-laser vision correction (LVC) ectasia were evaluated with SD-OCT and compared with changes observed in post-LVC eyes that were stable. The retrospective case-control study included 111 eyes from 111 stable post-LVC patients (41 trans-PRK, 34 LASIK, and 36 SMILE) and 23 patients who had post-LVC ectasia (1 trans-PRK, 1 sub-Bowman keratomileusis, 1 laser-assisted subepithelial keratomileusis, and 20 LASIK). A “unique epithelium remodeling pattern was observed” in both groups (stable and ectatic eyes). The authors reported that ectatic eyes had a “horseshoe-like pattern.” The authors also noted that the “top 3 ranked [area under the curve] [receiver operating characteristic] SD-OCT parameters were all derived from epithelium, including epithelium 5 mm superior-inferior (cutoff value = −0.675, AUC = 0.956), epithelium 5 mm inferior (cutoff value = 52.41, AUC = 0.937), and epithelium thickness range (cutoff value = −23.685, AUC = 0.936).” The authors concluded that the remodeling pattern and the SD-OCT parameters describing the epithelial remodeling could be useful for ectasia diagnosis. The study is published in the Journal of Cataract & Refractive Surgery.

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