Photo: Faith Ninivaggi
UMass Chan Medical School has entered a nonexclusive license agreement with Hongene Biotech Corporation to produce extended nucleic acid (exNA) monomers and exNA-modified oligonucleotides for research use.
Developed by Ken Yamada, PhD, assistant professor of RNA therapeutics, and Anastasia Khvorova, PhD, the Remondi Family Chair in Biomedical Research and professor of RNA therapeutics, exNA is a proprietary backbone modification that significantly enhances the durability and pharmacokinetics of oligonucleotide therapeutics, while maintaining compatibility with established small interfering RNA (siRNA) designs.
The exNA chemical scaffold stabilizes, sustains and extends the longevity of oligonucleotide drugs. The improved performance of exNA oligonucleotides has the potential to advance RNA-based translational research and achieve therapeutic success in living tissue, especially cells other than liver cells.
“We’re excited to be partnering with Hongene Biotech to make exNA oligonucleotide technology more widely available to scientists and investigators,” said Dr. Yamada. “Hongene’s expertise in phosphoramidite manufacturing and oligonucleotide synthesis allows us to expand access to exNA oligonucleotide technology for translational investigators.”
Oligonucleotide drugs, including siRNAs, are a new class of medicine that enables modulation of disease-causing genes for therapeutic effect. There are seven siRNAs approved for clinical use by the Food and Drug Administration—all targeting the liver—with more in late-stage clinical trials.
Under the terms of the agreement, Hongene will apply its expertise in phosphoramidite manufacturing and oligonucleotide synthesis to make exNA phosphoramidites and custom exNA oligonucleotides available through its catalogue and services.
The expanded access to exNA technology is expected to help advance research into more stable, tissue-specific RNA therapeutics and support the development of novel medicines targeting areas of unmet clinical need.
David Butler, PhD, chief technology officer at Hongene, added, “This partnership reflects our strategy to bring next-generation RNA chemistries to market and support researchers working on the toughest delivery challenges in oligonucleotide therapeutics.”
“By enabling access to exNA for research, we hope to accelerate the development of RNA-based medicines for extrahepatic indications,” said Dr. Butler.
