Annually in the U.S., about 40,000 people with corneal endothelial cell dystrophy receive a corneal transplant. The ligand of melanocortin MC1 receptor (MC1R) has been shown to protect the corneal endothelial cells from stress and injury in several models.
Previous studies have linked variants of complement factor H (CFH) and its alternative splicing isoform, FHL-1, with increased complement activation and risk of age-related macular degeneration (AMD).
A collaboration between Plaquetec Ltd. and a lab at the Babraham Institute has demonstrated the druggability of a pro-inflammatory protein discovered by Plaquetec.
Centogene NV and Evotec SE have announced the discovery of a new small molecule with potential to treat patients with type 2 and type 3 Gaucher disease, or neuronopathic Gaucher disease.
Ascidian Therapeutics Inc. recently provided preclinical data for ACDN-01, an AAV-encoded RNA exon editor targeting ABCA4, being developed for the treatment of ABCA4-related retinopathies, including Stargardt disease.
Arvinas Operations Inc. has synthesized proteolysis targeting chimera (PROTACs) compounds comprising an E3 ubiquitin ligase-binding moiety coupled to a B-cell lymphoma 6 protein (BCL6)-targeting moiety through a linker.
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