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).
Sitala Bio Ltd. has prepared new indole derivatives acting as complement factor B (CFB) inhibitors. They are thus reported to be useful for the treatment of age-related macular degeneration, schizophrenia, atypical hemolytic uremic syndrome, membranous nephropathy, myasthenia gravis, paroxysmal nocturnal hemoglobinuria, diabetic retinopathy and rheumatoid arthritis, among others.
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.
Gemvax & Kael Co. Ltd. has identified peptides reported to be useful for the treatment of age-related macular degeneration (AMD), cerebral amyloid angiopathy and Alzheimer’s disease.
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.
Day two of the J.P Morgan Healthcare Conference rolled on with positive data from Sling Therapeutics Inc. that is leading the company to a phase III study in treating thyroid eye disease. The privately held company posted top-line efficacy and safety results from a phase IIb/III study of its lead candidate, linsitinib, which hits its primary endpoint with statistical significance at the twice-daily, 150-mg oral dose.
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.
What does it mean for a confirmatory trial to be “underway”? That’s a question that’s been plaguing some drug sponsors, especially those in the ultra-rare disease space, since the U.S. Congress, in 2023, gave the FDA the authority to require that a confirmatory trial be underway at the time accelerated approval is granted.
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