报告人：Prof. Stefaan C. De Smedt, Editor-in-Chief Journal of Controlled Release
报告题目：Observations which might inspire drug delivery scientists for guiding bio-therapeutics over biological barriers
报告时间：2024年9月20日（周五）下午14:00-15：00
报告地点：909-B厅
个人简介：
Career at Ghent University. Dr. De Smedt studied pharmacy at Ghent University (Belgium). He graduated from Ghent University in 1995 and joined the pharmaceutical development group of Janssen Research Foundation. Since 1997 he has been a post-doctoral fellow of the Flemish Fund for Scientific Research at the Departments of Pharmacy of respectively Ghent University and the University of Utrecht (the Netherlands). In October 1999 he became Professor in Physical Pharmacy and Biopharmacy at Ghent University where he initiated research on advanced delivery of biologics/nanomedicines and founded the Ghent Research Group on Nanomedicines. Dr. De Smedt served as dean of the Faculty of Pharmaceutical Sciences at Ghent University from 2010 till 2014. He has been a member of the Board of Directors of Ghent University (2014-2022) and the Board of Directors of Ghent University Hospital (2019-2022). At Ghent University he has facilitated and promoted major research initiatives like the installation of the Biophotonic Research Group (Faculty of Pharmaceutical Sciences, 2007) and, more recently, the interdisciplinary consortia ‘RARE-Med’ (on precision medicine in rare diseases, 2019) and ‘Leveraging Biopharmaceutical R&D at UGent’ (2022).
Career as guest professor. In 2007 Dr. De Smedt has been Guest Professor at the Catholic University of Leuven (Belgium) and from 2012 till 2014 he was a Guest Professor at the University of Antwerp (Belgium). He has been Guest Professor at the Shanghai Jiao Tong University (2016-2017), Distinguished Visiting Scientist of the Chinese Academy of Sciences at the University of Science and Technology of China (Hefei 2017), Specially Appointed Professor of Nanjing Forestry University (since 2016) and Adjunct Professor at the University of Science and Technology of China (Hefei, 2020).
Editorships. Since 2004 Dr. De Smedt serves as the European Associate Editor of the Journal of Controlled Release (JCR), being a leading journal in Pharmacology & Pharmacy; In 2015 he became Editor of JCR (for the region Europe- the Middle East & Africa); Since 2020 he is the Deputy Editor-in-Chief of JCR; From January 2023 onwards he leads Journal of Controlled Release as Editor-in-Chief.
Member of scientific academies. He is a member of the Flemish Royal Academy of Medicine (elected 2015), the European Academy of Sciences (section medicine & life sciences; elected 2018) and the Académie Nationale de Pharmacie de France (elected 2019).
报告摘要：
Over the past 30 years protein bio-therapeutics experienced major clinical successes and nowadays represent the fastest growing category of drugs. Meanwhile, progress in the understanding of RNA biology has led to the identification of new RNA classes with unanticipated functions. Following these advances new therapeutic strategies based on nucleic acids have been introduced. Examples are single stranded antisense oligonucleotides, double stranded small interfering RNAs and microRNAs, all for inhibiting gene expression and, most recently, single guide RNAs for the editing of mutated genes. In addition to nucleic acids which silence or edit genes, there is a major interest as well in the therapeutic potential of messenger RNAs to express proteins in cells for e.g. vaccination to protect the population against infections or to treat cancer patients. As 30-40 years ago, when recombinant proteins and monoclonal antibodies began to be developed as therapeutics, it seems that with nucleic acid bio-therapeutics we are again on the brink of a revolution in drug development. Today the majority of clinically advanced nucleic acids are systemically injected and tested for liver associated diseases. However, numerous potential targets for nucleic acids are present in tissues beyond the liver, like in the lungs and the eyes, which can best be reached through local administration of the nucleic acids, like through inhalation in the lungs or local injections in the eye.
Fulfilling the therapeutic expectations of nucleic acids will however require to overcome major delivery hurdles, including how to keep them stable and how to deliver them across cellular, extra- and intracellular membranes into the cytosol. Currently, the packaging of nucleic acids into well designed nanoparticles (‘nanomedicines’) is the major approach taken to deliver them in cells. While major succeses have been achieved, it remains however of paramount importance not to forget that only a very low percentage (~ 1%) of systemically injected nucleic acids packaged in nanoparticles become successfully delivered at their targets. The lack of safe and efficient delivery technologies remains the major bottleneck for the breakthrough of nucleic bio-therapeutics.
At Ghent University we undertake efforts for a better understanding of the physicochemical and biopharmaceutical understanding of nanomedicines, including lipid nanoparticles (LNPs) loaded with nucleic acids, this to improve their delivery performance and safety. Though, while the clinical introduction of mRNA vaccines has boosted the interest and investments by the pharmaceutical industry into the further development of LNPs, at university we consider it our role to keep on trying to identify new concepts and strategies which might open new avenues for the delivery of bio-therapeutics to targets which cannot be reached through nanoencapsulation. This lecture will explain our recent efforts in finding strategies to deliver nucleic acids from the extracellular space, over cell- and organelle-membranes, into the cytosol. Both pharmaceutical, biological, physical and engineering aspects of our work will be highlighted. A special emphasis will be on the delivery of nucleic acids to targets in the eye.
联系人：殷黎晨教授