(29b) Keynote Speaker - Amit Khandhar, Director of Formulations, HDT Bio

Messenger RNA (mRNA) vaccines are leading the fight against the ongoing COVID-19 pandemic. The adaptability of the mRNA platform allows for rapid updating of current vaccines to target emerging SARS-CoV-2 variants that escape vaccine-induced immunity. The urgent need to stem a global pandemic was the key stimulus to drive widespread adoption of mRNA vaccines, but several technological innovations over decades of development converged to enable the success we see today. One of these technological advancements includes the use of lipid nanoparticle (LNP) formulations to protect and deliver the chemically labile mRNA molecules to host cells. However, LNP-formulated mRNA vaccines have a very short shelf-life and must be stored at ultra-low temperatures (-80°C), requiring an extensive freezer infrastructure to maintain vaccine stockpiles — this has significantly impacted global distribution in resource limited regions. Moreover, the inherent nature of mRNA encapsulation in LNPs will make current vaccine stockpiles obsolete when next-generation vaccines targeting circulating variants enter the pipeline. We developed a two-vial system that combines self-amplifying RNA (saRNA) technology with a refrigerator-stable oil-in-water emulsion-based formulation called LION (Lipid InOrganic Nanoparticle). The LION formulation is amenable to production at > 100 M doses/day and can be stockpiled at 2–8°C for years. As new generations of mRNA or saRNA vaccines enter the pipeline, the LION stockpile can be distributed to support global vaccination efforts by simple on-site mixing with the RNA vaccine product. Alternatively, RNA products can be premixed with LION, lyophilized, and stored long-term at 2–8°C, providing a ready stockpile of RNA-based countermeasures targeting a range of pandemic-potential pathogens. Our saRNA/LION SARS-CoV-2 vaccine has now completed phase I clinical trial evaluation in India, under the drug designation “HGC019” and entered phase I evaluation in Brazil, South Korea, and the United States under the designation “HDT-301.” The development of thermostable RNA products will ultimately play a central role in equitable dissemination of a highly adaptable technology that can effectively curb future pandemics through global access.

Speaker Bio:

Amit is a materials scientist with a background in developing nanoparticle formulations for vaccines and drug delivery. He earned his PhD from the University of Washington in Seattle from where he co-founded a biotech company to commercialize nanoparticle tracers for novel magnetic imaging modalities. From there he migrated to infectious diseases research, where he collaborated with scientists in virology and immunology to develop next-generation nanoparticle-based adjuvants and delivery vehicles. At HDT Bio, Amit is leading the effort of thermostable RNA vaccine products.