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.
Entrada Therapeutics Inc. has updated progress across its preclinical portfolio of RNA-based programs for the potential treatment of neuromuscular and ocular diseases.
Guangzhou Runer Ophthalmic Biotechnology Co. Ltd. has identified complement factor B (CFB) inhibitors reported to be useful for the treatment of age-related macular degeneration, asthma, atherosclerosis, Crohn’s disease, glomerular disorders, hemolytic uremic syndrome, neurological disorders and diabetic retinopathy, among others.
Viatris Inc. has announced FDA clearance of its IND application for MR-146, an Enriched Tear Film (ETF) AAV gene therapy candidate for the treatment of neurotrophic keratopathy. The company plans to initiate a phase I/II trial in patients with neurotrophic keratopathy in the first half of next year.
Coave Therapeutics SA has nominated a lead gene therapy program, CoTx-101, for the treatment of retinal vascular diseases, such as wet age-related macular degeneration and diabetic macular edema.
Innovstone Therapeutics Ltd. has disclosed integrin αLβ2 (LFA-1)/ICAM-1 interaction inhibitors and/or RASP-trapping agents reported to be useful for the treatment of dry eye syndrome.
Researchers at Carl von Ossietzky Universität Oldenburg generated retinal pigment epithelial cell lines lacking three RPGR isoforms and examined whether they showed the defects in ciliary structure and actin turnover known to occur in retinitis pigmentosa.
RSS
