Gene therapies employing genetic drugs such as small interfering RNA (siRNA) for gene silencing and mRNA for gene expression have the potential to cure most diseases. However, sophisticated delivery systems are required to enable clinical use of nucleic acid polymers as they are readily broken down in biological fluids, do not accumulate at sites of disease and cannot penetrate 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 polymer in well-defined nanoparticles that protect the nucleic acid payload in vivo and facilitate intracellular delivery following uptake into target cells by endocytosis. This approach has received clinical validation with the approval of Onpattro by the FDA in 2018. Onpattro consists of an LNP containing siRNA to silence transthyretin in hepatocytes, thereby arresting and reversing the disease transthyretin induced amyloidosis (hATTR), a disease that was previously untreatable and was fatal within five years of diagnosis. In this talk I will describe the design features that were followed to develop Onpattro and how related technology is being employed to enable mRNA-based drugs. A notable example is the development of the Pfizer/BioNTech mRNA vaccine, which is playing a leading role in alleviating the Covid-19 pandemic.
Dr. Pieter R. Cullis
Department of Biochemistry and Molecular Biology
University of British Columbia
Scientific Director & CEO
Nanomedicines Innovation Network
Canada's National Centre of Excellence for NanoMedicines
Dr. Cullis and colleagues have been responsible for the development of nanomedicines employing lipid nanoparticle (LNP) delivery technology leading to five clinically approved drugs for cancer therapies, gene therapies and vaccines. Two recent examples are Onpattro (the first approved RNAi drug) to treat the hereditary condition transthyretin-induced amyloidosis and BNT162b2, the COVID-19 mRNA vaccine marketed by Pfizer/BioNTech. Dr. Cullis has co-founded ten biotechnology companies that now employ over 300 people, has published over 350 scientific articles and is an inventor on over 60 patents.
The William and Carmela Riker Memorial Lecture Series
This endowed fund was established in 1981 to honor the memory of Carmela Louise Riker, the wife of Dr. William K. Riker, chair of Pharmacology at OHSU for 21 years. Through her activities in the Medical School Alliance and OHSU Volunteer Services, Mrs. Riker made important contributions to enhancing patient facilities and supporting medical students. Following the death in 2004 of William Riker, the Riker family and the Department of Physiology and Pharmacology decided to honor his memory by renaming the fund. This lectureship supports an annual invited lecture by a distinguished scientist and educator in the field of pharmacology. The annual lectures are supported by the income from The William K. and Carmela L. Riker Memorial Lecture Fund of the OHSU Foundation. The Lecture Fund was initially established through the many generous donations of friends, family, colleagues and organizations. The Lecture Fund principal continues to grow because of ongoing donations in Dr. and Mrs. Riker's memory. Contributions may be made to the OHSU Foundation William K. and Carmela L. Riker Memorial Lecture Fund.
For additional information please contact Amy Johnson.