Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular and Clinical Ophthalmology Basel and collaborators proposed a novel dual AAV vector system to deliver a split-intein adenine base editor for precise correction of the most common Stargardt mutation, c.5882G>A (p.Gly1961Glu).

Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the treatment of optic neuropathy in familial dysautonomia and BPN-36964 for systemic treatment of familial dysautonomia.

Inherited retinal diseases (IRDs) are a group of monogenic disorders caused by mutations in more than 280 genes, and are characterized by progressive degeneration of photoreceptor cells in the retina.

Corneal neovascularization (CNV) occurs when abnormal blood vessels grow into the cornea in response to ischemic or hypoxic conditions caused by infections, inflammation or chemical injuries.

Pulsesight Therapeutics SAS has submitted a clinical trial authorization (CTA) to the French authority ANSM seeking to conduct a first-in-human phase I trial of PST-611 for dry age-related macular degeneration (AMD)/geographic atrophy (GA).

The company is advancing a pipeline of therapeutics, including programs acquired from Denali Therapeutics Inc., focused on treating neurological, cardiometabolic and ophthalmic diseases.

Proper survival and regeneration of retinal ganglion cells (RGCs), which constitute the optic nerve, is crucial for managing traumatic or ischemic optic nerve injury, optic neuropathy, or optic pathway gliomas. Researchers from Wenzhou Medical University described the synthesis of novel spirocyclic chalcone derivatives designed to combat optic nerve damage.