Over Engineering nanotechnology to unleash RNA therapeutics’ full potential

Engineering nanotechnology to unleash RNA therapeutics’ full potential

Avondlezing door dr. Roy van der Meel, georganiseerd door de Bossche Chemische Kring.

De lezing wordt voorafgegaan door de Algemene Ledenvergadering (aanvang 19.45 uur).

Samenvatting
Nucleic acid therapeutics harbor great potential for silencing, expressing, or editing genes. However, nucleic acid-based drugs require chemical modifications and sophisticated nanotechnology to prevent their degradation, reduce immunostimulatory effects, and ensure intracellular delivery [1].
Lipid nanoparticle (LNP) technology is the current gold standard delivery platform technology that has enabled the clinical translation of the first siRNA drug Onpattro [2] and the COVID-19 mRNA vaccines [3].
Nevertheless, currently approved LNP systems are mostly suited for vaccine purposes following local administration or hepatic delivery following intravenous administration [4].
To unleash RNA therapeutics’ full potential, nanotechnology with tuable biodistribution needs to be established that enables safe and delivery of nucleic acid drugs following systemic administration.
During this talk, I will discuss LNP delivery technology and focus on important aspects that have enabled its clinical translation such as development of ionizable cationic lipids. In addition, I will provide examples of therapeutic applications and highlight developments beyond the-state-of-the-art including engineering nature-inspired nanoplatform technology for systemic nucleic acid delivery to immune cells using apolipoproteins [5].

References

1. Kulkarni JA, Witzigmann D, Thomson SB, Chen S, Leavitt BR, Cullis PR, van der Meel R. The current landscape of nucleic acid therapeutics. Nat Nanotechnol. 2021 Jun;16(6):630-643. doi: 10.1038/s41565-021-00898-0.
2. Akinc A, Maier MA, Manoharan M, Fitzgerald K, Jayaraman M, Barros S, Ansell S, Du X, Hope MJ, Madden TD, Mui BL, Semple SC, Tam YK, Ciufolini M, Witzigmann D, Kulkarni JA, van der Meel R, Cullis PR. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nat Nanotechnol. 2019 Dec;14(12):1084-1087. doi: 10.1038/s41565-019-0591-y.
3. Verbeke R, Lentacker I, De Smedt SC, Dewitte H. The dawn of mRNA vaccines: The COVID-19 case. J Control Release. 2021 May 10;333:511-520. doi: 10.1016/j.jconrel.2021.03.043.
4. Kulkarni JA, Witzigmann D, Chen S, Cullis PR, van der Meel R. Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics. Acc Chem Res. 2019 Sep 17;52(9):2435-2444. doi: 10.1021/acs.accounts.9b00368.
5. Hofstraat SRJ, Anbergen T, Zwolsman R, Deckers J, van Elsas Y, Trines MM, Darwish YB, Priem B, Kleuskens T, Versteeg I, Maas R, Borges F, Verhalle LM, Tielemans W, Vader P, de Jong O, Teunissen AJP, Brechbühl E, Janssen HM, Fransen PM, de Dreu AD, Schrijver DP, Toner YC, Beldman TJ, Netea MG, Mulder WJM, Kluza E, van der Meel R. A nature-inspired nanodelivery platform for gene silencing in hematopoietic stem and progenitor cells. Research Square. 2023. doi: 10.21203/rs.3.rs-3458536/v1

CV
Roy van der Meel is a biomedical engineer specialized in nanomedicine and RNA therapeutics.
After obtaining a PhD from Utrecht University under guidance of prof. dr. Wim Hennink and prof. dr. Gert Storm, and a postdoc appointment at the University Medical Center Utrecht in prof. dr. Raymond Schiffelers’ lab, he secured Marie Skłodowska-Curie and Dutch Research Council (NWO) Veni funding to move to prof. dr. Pieter Cullis’ lab at the University of British Columbia in Vancouver, Canada.
During his 3.5-year postdoctoral tenure, he gained extensive experience with developing lipid nanoparticle (LNP) technology that enabled the approval of the first siRNA therapeutic and the COVID-19 mRNA vaccines. 
In 2019, he was recruited to the Eindhoven University of Technology by prof. dr. Willem Mulder and appointed Assistant Professor in the Precision Medicine group. 
His current research is supported by NWO Vidi funding and focuses on establishing platform nanotechnology for delivering RNA therapeutics to specific immune cells. 
He has co-authored over 50 publications in leading nanotechnology-focused journals including Nature Nanotechnology, Nature Biomedical Engineering, and ACS Nano.

Web/socials

• https://www.mulderlab.com/
• https://www.tue.nl/en/research/researchers/roy-van-der-meel
• https://scholar.google.com/citations?user=4brXEMMAAAAJ&hl
• https://twitter.com/rvdmnanomeds
• https://www.linkedin.com/in/royvandermeel

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