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lufepirsen (OcuNexus Therapeutics)
- Development stage
- Unknown
- Lead developer
- Glaukos
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Ophthalmic
01
Overview
Lufepirsen is an antisense oligonucleotide drug formulated as a thermoreversible ophthalmic gel, specifically designed to treat persistent corneal epithelial defects (PCED). Originally developed by OcuNexus Therapeutics and currently being advanced by Amber Ophthalmics, the drug's mechanism of action involves the downregulation of Connexin-43 (Cx43), a gap junction protein that becomes overexpressed following ocular injury. By reducing Cx43 levels, lufepirsen limits the formation of hemichannels, thereby quenching unregulated inflammation and promoting the migration of corneal epithelial cells to close the defect. It has demonstrated efficacy in clinical trials for PCED resulting from various etiologies, including chemical and thermal burns, and has been granted Orphan Drug Designation by the FDA.
- Brand names
- Nexagon
- Other names
- lufepirsen sodium
02
Targets
GJA1 (Connexin43 Hemichannel)
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