Patients with Leber hereditary optic neuropathy who received bilateral injections of Gensight Biologics SA’s Lumevoq (lenadogene nolparvovec) are continuing to see statistically significant visual improvements three years into the phase III REFLECT trial, but the missed primary endpoint at 1.5 years, along with a series of manufacturing mishaps, have left the gene therapy’s approval prospects uncertain.

Abeona Therapeutics Inc. has announced three investigational preclinical gene therapy product candidates from its ophthalmology program. The new AAV-based therapies use novel AAV capsids from Abeona's in-licensed AIM capsid library. Abeona intends to submit its first pre-IND application meeting request this month.

Novelmed Therapeutics Inc. has announced that NM-3086, the lead clinical asset in its properdin-associated alternative pathway (AP) program...

Eyebiotech Ltd. has announced details on...

Praetego Inc. has been granted a US$300,000 phase I Small Business Technology Transfer Research (STTR) award by the National Eye Institute (NEI) to advance the company's lead candidate, PTG-630, into preclinical proof of concept in diabetic retinopathy.

Ophthalmic startup Eluminex Biosciences Ltd. closed a $40 million series B round to progress its pipeline of ophthalmic assets and recombinant human collagen technology. Eluminex’s pipeline includes multi-targeted antibody molecules for vision-threatening retinal diseases and an oral small molecule for rare inherited pediatric retinal dystrophies, but its lead asset, EB-301, is a biosynthetic cornea derived from recombinant human type III collagen.

Aldeyra Therapeutics Inc. is eyeing the start of clinical trials with reactive aldehyde species (RASP) modulators ADX-246 for systemic immune-mediated diseases and ADX-248 for geographic atrophy.

After a delay in November that resulted in a new PDUFA date, Apellis Pharmaceuticals Inc.’s Syfovre (pegcetacoplan injection) received U.S. FDA approval for treating geographic atrophy (GA) secondary to age-related macular degeneration. While this is the first and only FDA-approved treatment for GA, there is competition afoot from Belite Bio Inc. and Iveric Bio Inc. The intravitreal targeted C3 therapy’s Feb. 17 approval of the priority NDA came more than a week ahead of its Feb. 26 PDUFA.

Akari Therapeutics plc has advanced its preclinical development program for long-acting PAS-nomacopan for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). The company has produced new and promising versions and progressed from lab scale to pre-GMP optimization, and is targeting an IND application to the FDA in the first half of next year.

Retinal disorders such as diabetic macular edema (DME), wet age-related macular degeneration (wAMD) or uveitic macular edema share inflammatory traits mediated by vascular endothelial growth factor (VEGF) and interleukin-6 (IL-6), among others.