Neurophth Therapeutics Inc. has received FDA clearance of its IND application for the in vivo gene replacement therapy NFS-02, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized NADH-dehydrogenase subunit 1 (ND1) gene, for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND1 mutation.
Aviceda Therapeutics LLC has completed IND-enabling good laboratory practice (GLP) toxicity studies of its lead ophthalmic drug candidate, AVD-104, in nonhuman primates and rabbits.
Sparingvision SAS has obtained FDA clearance for its IND application for SPVN-06, its lead gene-independent therapy for the treatment of retinitis pigmentosa (RP). SparingVision has also submitted a clinical trial authorization (CTA) application to the French regulator (ANSM), which is currently under review.
Opus Genetics Inc. has received FDA clearance for its IND application for a first-in-human phase I/II trial of OPGX-001 in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5). The trial is due to start in the U.S. early next year (ClinicalTrials.gov Identifier NCT05616793).
Unicycive Therapeutics Inc. has announced key efficacy findings with UNI-494 in animal models of geographic atrophy (GA). UNI-494 is a mitochondrial potassium channel (mitoKATP) activator in development for treating diseases affected by mitochondrial dysfunction, such as kidney, liver and ocular diseases.
Aerie Pharmaceuticals Inc. has divulged dual-acting compounds consisting of a glucocorticoid moiety and a Rho kinase 1 (ROCK) inhibitor bound through a linker. They are reported to be useful for the treatment of eye disorders.
Merck Sharp & Dohme Corp. has disclosed plasma kallikrein (KLKB1) inhibitors reported to be useful for the treatment of hereditary angioedema, diabetic macular edema, retinal vein occlusion, wet macular degeneration (exudative) and uveitis.
Primary angle closure glaucoma (PACG) is characterized by anatomic blockage of the drainage angle of the eye leading to elevated eye pressure and optic neuropathy, and its molecular pathogenesis is still not well studied.
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