Cancer Focus Fund LP is investing US$5 million in funding to support ISA Pharmaceuticals BV's ISA-103 in a first-in-human study for the treatment of uveal melanoma.
Age-related macular degeneration (AMD) is the leading cause of irreversible vision loss in persons over the age of 50. While poorly understood, AMD pathogenesis begins with structural disorganization of the outer-blood-retina-barrier (oBRB), containing retinal pigment epithelium (RPE) and associated Bruch’s membrane.
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).
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.
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