Guangzhou Runer Ophthalmic Biotechnology Co. Ltd. has divulged compounds acting as complement factor B (CFB) inhibitors reported to be useful for the treatment of age-related macular degeneration, diabetic retinopathy, uveitis, retinitis pigmentosa, atypical hemolytic uremic syndrome, macular edema, membranous glomerulonephritis and paroxysmal nocturnal hemoglobinuria (PNH).
Disruption of alternative splicing can generate abnormal mRNA variants, producing nonfunctional proteins or triggering nonsense-mediated decay, and is implicated in many splicing-related diseases. Therapeutic approaches that modulate splicing, therefore, hold promise to redirect aberrant transcripts toward normal isoforms and reestablish functional protein expression.
Fannin Partners LLC has been awarded a $300,000 phase I Small Business Innovation Research (SBIR) grant from the National Eye Institute (NEI) to advance a first-in-class, non-VEGF-targeted therapeutic for wet age-related macular degeneration (AMD) in patients not responding to anti-VEGF therapies.
