ProteinQure announced new preclinical results today, demonstrating efficient delivery of small interfering RNA (siRNA) to the central nervous system (CNS) in non-human primates. Using intrathecal administration of proprietary peptide-oligonucleotide conjugates that leverage receptor-mediated transport, the study provides evidence of widespread distribution of siRNA throughout the brain.

The data establishes ProteinQure’s platform as competitive with best-in-class approaches. In the 28-day study, the ProteinQure conjugates were directly compared to lipid-based approaches that are considered leading CNS delivery platforms in clinical development. “These results highlight the potential of our siRNA conjugate technology to overcome long-standing barriers in nucleic acid delivery to the brain,” said Lucas Siow, CEO of ProteinQure. “By coupling optimized peptides to oligonucleotides, we have achieved broad CNS biodistribution in primates, with superiority in certain cell types — a critical step toward advancing oligonucleotide therapeutics for neurological disorders.”

These findings and additional early data on ProteinQure’s blood-brain barrier shuttle program will be presented at the 21st Annual Meeting of the Oligonucleotide Therapeutics Society in Budapest, October 2025.

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