The Future of Genomic Medicine
Developing lipid nanoparticle delivery systems laid the groundwork for COVID-19 vaccines and the future of medicine
Jointly presented by the HIBAR Research Alliance and the National Academy of Inventors
During the record-breaking sprint to develop mRNA COVID-19 vaccines, developers faced a key challenge: they needed a drug delivery system that would enable the messenger RNA, or mRNA, to get into cells in the body. Fortunately, this was not the first time this challenge had been encountered, because the delivery of RNA or DNA to cells to prevent or treat disease at its genetic root cause has been under development for decades, and the success of the COVID-19 vaccine is the tip of the iceberg for genetic medicine.
This delivery solution uses lipid nanoparticles – microscopic bubbles of fat – to encase and protect the mRNA and enable it to be taken up by a cell and released inside to produce the desired immune response. Decades of research in lipid systems enabled the dramatic speed of the COVID-19 vaccine development, from concept to clinical trials within 3 months of sequencing the viral genome. The groundwork for lipid nanoparticle delivery systems was laid more than 40 years ago in the lab of Dr. Pieter Cullis at the University of British Columbia in Vancouver. Since then, he has played a founding role in a number of companies developing pharmaceutical solutions based on drug delivery research, including Inex Pharmaceuticals, Acuitas Therapeutics, and Precision Nanosystems.
Prior to a year or so ago, lipid nanoparticles were relatively unknown, despite the decades of effort by many researchers to demonstrate their value as drug delivery systems. As a result of the demonstrated success of the COVID-19 vaccine, lipid nanoparticle–RNA drugs are now poised to cause a revolution in medicine, because of their ability to deliver precision gene therapy drugs to treat a wide range of diseases, and to enable vaccines for many other infectious diseases.
University of British Columbia
Collaborators Dr. Pieter Cullis (Professor, University of British Columbia) and Dr. James Taylor (Co-founder and CEO, Precision Nanosystems) described the HIBAR research journey that led to the global vaccine success, the prospects for a revolution in genomic medicines, and some of the HIBAR research challenges they faced along the way.