PANEL DISCUSSION ON OLIGONUCLEOTIDES AND THERAPEUTICS
BRUCE H MORIMOTO, PhD
Vice President, Drug Development, Alto Neuroscience, Inc.
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TRENDS, CHALLENGES, AND FUTURE DIRECTIONS
As I thaw out of nearly three years of pandemic-induced disruption of daily life, I reflect on the journey that oligonucleotide therapeutics have taken us. Pre-pandemic, my thoughts of nucleic acid therapeutics were limited to anti-sense oligonucleotides (ASO) and siRNA, although considerable interest existed in mRNA-therapeutics, my exposure to mRNA vaccines was for rabies, Zika, and Chikungunya viruses.
This all changed at the end of 2019 when the global explosion of COVID-19 forced the pharmaceutical industry to rapidly develop a vaccine. Traditional vaccine development relied on either attenuated virus or protein antigens to induce an immune response. Sequencing of SARS-CoV2, the virus that causes COVID-19 allowed for a new class of mRNA vaccine. But this is only part of the story as mRNA is chemically unstable. The parallel development of lipid nanoparticles (LNP), starting around 2001 for the delivery of D ...
