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Solving a 20-year-old mystery could lead to hundreds of new ASO medicines

Why are some antisense drugs better tolerated than others? It’s a question that’s vexed researchers for more than two decades. Ionis’ persistence in finding the answer has led to a breakthrough that could potentially bring hundreds of new drug candidates to the clinic and ultimately lead to more treatments for patients with diseases previously not treatable with RNA-targeted therapy.

The results of their paradigm-shifting research, recently published in Nature Biotechnology, are creating a buzz in the scientific community. “The paper is important because it provides a step-by-step, testable mechanism to explain the behaviors of ASOs. It provides an immediate solution to the problem of poorly tolerated ASOs, meaning many more very potent ASOs that would otherwise be discarded can now be used, and it should reduce the number of ASOs that need to be screened to identify potent and safe drug candidates,” said Punit P. Seth, Ph.D., vice president, Medical Chemistry at Ionis and one of the senior authors of the paper.

Of the groundbreaking research, Dr. Seth added, “Along with efforts to enhance productive delivery, this work opens a new horizon for antisense technology to bring even better antisense medicines to patients. It once again demonstrates the value of basic research in advancing what still is a relatively nascent technology, RNA- focused drug discovery.”