Week in review: Gene therapies, steroid potential, sanitizer hazards

A weekly roundup of ophthalmic news from around the web.

The FDA has awarded fast-track designation to a gene therapy candidate for treatment of achromatopsia due to mutations in CNGA3. Developed by MeiraGTx and Janssen Pharmaceuticals, AAV-CNGA3 is administered to cone receptors via subretinal injection to restore cone function. The treatment has already been granted orphan drug designation by both the FDA and EMA, and was designated as a drug for rare pediatric disease by the FDA. An open-label, dose-escalation phase 1/2 clinical trial is underway. MeiraGTx

Meanwhile, an optogenetics-based gene therapy for Stargardt disease was granted orphan drug status, according to Nanoscope Therapeutics. "We are excited by the potential of ambient light activatable Multi-Characteristic Opsin (MCO) based photosensitization of retinal neurons for treating Stargardt disease in a gene agnostic manner," explained Nanoscope’s chief executive officer Sulagna Bhattacharya of their treatment, vMCO-010. Since Stargardt disease shares characteristics with dry AMD, the company hopes that lessons from this program will help spur MCO-based therapies for dry AMD. Nanoscope Therapeutics

New data highlight the potential of a unique treatment for post-cataract surgery pain and inflammation. The treatment is a formulation of 0.2% betamethasone—the first U.S. ophthalmic therapeutic to utilize the potent corticosteroid—delivered using Surface Ophthalmics’ patented Klarity vehicle that the company claims will improve efficacy and safety. Phase 2 results demonstrate that patients receiving 2 daily doses of SURF-201 were more likely to exhibit clearance of anterior chamber cells at day 8 and 15 than what has been previously observed with other branded corticosteroids. Furthermore, nearly 90% of patients were pain free at day 15—an unprecedented rate, according to the company. The treatment appeared safe and was well tolerated. Surface Ophthalmics

An unexpected danger of hand sanitizers? Pediatric eye injuries. According to a national review from the French Poison Control Centers, the increased use of alcohol-based hand sanitizers led to a 7-fold increase in ocular exposures in children between April and August 2020 when compared with 2019. The incidents often occurred in public places with hand sanitizer devices and included several cases of serious corneal lesions. The authors conclude that although alcohol-based hand sanitizers are important to mitigate spread of COVID-19, they should be used with care, particularly around young children. JAMA Ophthalmology

Faricimab continues to impress, meeting primary endpoints in a pair of phase 3 studies for neovascular AMD. Genentech announced that the novel VEGF inhibitor, administered at intervals of up to every 16 weeks, yields noninferior visual acuity gains to aflibercept dosed every 8 weeks. The treatment also achieved an unprecedented level of durability for an injectable AMD treatment in a phase 3 study, with 45% of patients being treated every 16 weeks in the first year. These results come on the heels of positive news demonstrating faricimab’s potential for DME. Genentech