Extended Nucleic Acid Therapeutics: Unlocking potential through tiny methylene chemical modifications

Dr. Ken Yamada is an Assistant Professor at the RNA Therapeutics Institute (RTI) at UMass Chan Medical School, where he develops novel chemistry platforms to enable oligonucleotide therapeutics in challenging extra-hepatic tissues. He received his BS, MS, and PhD from the Tokyo Institute of Technology, where he began his training in nucleic acid chemistry in Prof. Mitsuo Sekine’s lab. After his doctoral work, he continued advancing oligonucleotide chemistry through positions at McGill University, Tohoku University, and later in Prof. Anastasia Khvorova’s lab at RTI. At RTI’s highly collaborative “Powerhouse,” he and his colleagues have developed pioneering chemistry platforms that significantly enhance the PK/PD properties of siRNA therapeutics. Most notably, he invented extended nucleic acid (exNA), now incorporated into a lead siRNA entering Phase I clinical evaluation for CNS disorders and soon to be available commercially as phosphoramidites, with several of his innovations already licensed by major biotech companies.