Date：26 July (Wednesday)
Time：09:00 – 10:30 (GMT+8)
University of British Columbia
Pieter R. Cullis, PhD, FRSC, FNAI (USA), OC, Director, Nanomedicines Research Group, Professor, Department of Biochemistry and Molecular Biology, University of British Columbia. Dr. Cullis and co-workers have been responsible for fundamental advances in the development of nanomedicines employing lipid nanoparticle (LNP) technology for cancer therapies, gene therapies and vaccines. This work has contributed to five drugs that have received clinical approval by the FDA, the European EMA and Health Canada. Dr. Cullis has also co-founded eleven biotechnology companies that now employ over 400 people, has published over 350 scientific articles (h index 133) and is an inventor on over 100 patents. He has also co-founded and been Founding Scientific Director of two National Centre of Excellence networks, the Centre for Drug Research and Development (now AdMare) in 2004 and the NanoMedicines Innovation Network in 2019. These not-for-profit networks are aimed at translating basic research in the life sciences into commercially viable products and have given rise to numerous start-up companies. Dr. Cullis has received many awards including the Order of Canada in 2021 and the VinFuture Prize (Vietnam), the Prince Mahidol Award (Thailand), the Gairdner International Award (Canada) and the Tang Prize (Taiwan) in 2022. Two recently approved drugs that are enabled by LNP delivery systems devised by Dr. Cullis, members of his UBC laboratory and colleagues in the companies he has co-founded deserve special emphasis. The first is Onpattro which was approved by the US FDA in August 2018 to treat the previously fatal hereditary condition transthyretin-induced amyloidosis (hATTR). Onpattro is the first RNAi drug to receive regulatory approval. The second is Comirnaty, the COVID-19 mRNA vaccine developed by Pfizer/BioNTech that has received regulatory approval in many jurisdictions including Canada, the USA, the UK and Europe. Comirnaty is playing a major role in containing the global Covid-19 pandemic with approximately 6B doses administered worldwide in 2021 and 2022.
Synopsis: Gene therapies employing genetic drugs such as small interfering RNA (siRNA) for gene silencing and messenger RNA (mRNA) for gene expression have the potential to cure most diseases. However, sophisticated delivery systems are required to enable the therapeutic use of nucleic acid polymers as they are quickly broken down in biological fluids, do not accumulate at sites of disease and cannot penetrate into target cells even if they arrive at target tissues. Lipid nanoparticle (LNP) technology is increasingly enabling the clinical potential of genetic drugs by packaging the nucleic acid in well-defined nanoparticles that protect the payload following systemic in vivo administration and facilitate intracellular delivery following uptake into target cells by endocytosis. The first clinical validation of this approach was achieved by the approval of Onpattro by the US FDA in 2018 to treat the disease transthyretin-induced amyloidosis (hATTR. Onpattro consists of LNPs containing siRNA that silences production of transthyretin in the liver (in hepatocytes) following intravenous administration. In this talk I will describe the historical development of LNP systems leading to the development of Onpattro and how related LNP delivery technology is being employed to enable many mRNA-based gene therapy drugs. A notable example of the success of this approach is the development of Comirnaty, the Pfizer/BioNTech COVID-19 mRNA vaccine, which has played a leading role in alleviating the Covid-19 pandemic.