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).
Word that big pharma firms are sniffing around Horizon Therapeutics plc as a takeover candidate caused not only that firm’s shares to jump but also provided a boost for Viridian Therapeutics Inc., which has a thyroid eye disease (TED) candidate set to challenge Horizon’s Tepezza (teprotumumab-trbw), cleared by the U.S. FDA in January 2020.
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.
A lackluster efficacy signal has prompted Editas Medicine Inc. to pause enrollment in a phase I/II trial of its CRISPR/Cas9-based gene editing therapy, EDIT-101, which is in development for patients with a particular form of Leber congenital amaurosis type 10.
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.
Newco Breye Therapeutics ApS is poised to repurpose an intravenously administered drug that failed in ischemia reperfusion injury as an orally available treatment for diabetic retinopathy, after closing a €4 million (US$4.1 million) seed round.
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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