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Lufepirsen is an unmodified antisense oligonucleotide administered as an ophthalmic gel for the treatment of persistent corneal epithelial defects (PCED), particularly those secondary to trauma and refractory to standard care. It acts by inhibiting the expression of connexin 43 (GJA1), a cell membrane hemichannel-forming protein that is overexpressed after acute injury and chronic disease states. By targeting connexin 43, lufepirsen aims to restore limbal microvasculature and promote regeneration of the corneal epithelium. The drug has orphan designation for corneal injuries and is in Phase III clinical development for PCED. Lufepirsen was originally developed by CoDa Therapeutics and has been further developed by Amber Ophthalmics[1][2][4][7].
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