Translation, interrupted.
How exaluren works
Exaluren promotes premature termination codon (PTC) readthrough via selective ribosome modulation, partially restoring full-length protein production.
Nonsense mutation creates a premature stop codon (PTC)
A single nucleotide substitution converts an amino acid codon into a premature stop codon (PTC). Protein translation terminates early, producing a non-functional protein fragment — the root cause in NMAS, NM-ADPKD, and other nonsense mutation conditions.
Exaluren selectively modulates the human ribosome
Exaluren binds to the small (40S) subunit of the eukaryotic ribosome, reducing discrimination at the PTC — allowing a near-cognate aminoacyl-tRNA to insert and translation to continue.
Full-length functional protein restored
Translation completes, producing a full-length protein. Gene-agnostic mechanism: the same compound addresses nonsense mutations across COL4A3, COL4A4, COL4A5 (Alport) and PKD1/PKD2 (ADPKD).
Designed for selectivity and long-term administration
Administered subcutaneously. Markedly reduced affinity for mitochondrial and bacterial ribosomes versus aminoglycoside antibiotics. Concentrates in kidney cells via megalin-mediated uptake.
Designed for selectivity and kidney concentration
Human Ribosome Selectivity
Markedly reduced affinity for mitochondrial and bacterial ribosomes — designed to avoid the ototoxicity and nephrotoxicity associated with aminoglycoside antibiotics at therapeutic doses.
Kidney-Concentrated Delivery
Administered subcutaneously and transported into cells via megalin — a receptor highly expressed in kidney and epithelial tissues — supporting preferential accumulation in kidney cells.
Gene-Agnostic Platform
Targets the premature stop codon, not the gene — enabling investigation across multiple rare diseases caused by nonsense mutations from a single compound.