Explore the recording of our half-day virtual event, where leading voices in nucleic acid development, delivery, and manufacturing shared their expertise. Gain practical insights from experts shaping the future of gene expression modulation, gene editing, and nanoparticle-based drug development, including Dan Peer (Precision NanoMedicine), Heinrich Hass (NeoVac), and Jacek Lubelski (NanoCell).
Why Watch?
- Learn from Real-World Case Studies: Discover how experts apply scalable purification workflows, analytical innovations, modulate gene expression, and implement targeted delivery strategies.
- Solve CMC Challenges: Understand how to simplify analytical workflows, improve formulation predictability, and gain deeper insights into LNP drug products.
- Explore New Frontiers: Hear about novel RNA sequences and LNP technologies with potential applications in oncology, vaccines, and more.
- Network with Leaders: Connect with researchers, formulation scientists, and decision-makers driving progress in RNA and nanoparticle therapeutics.
Watch the recording to enhance your understanding and stay informed on the latest advancements in the field.
Session 1 | Nucleic Acid Delivery for Advanced Therapies
Revisit the first session of our virtual event, where experts from NIH, NeoVac, and Sartorius BIA Separations shared breakthroughs in nucleic acid delivery. This session covers scalable RNA manufacturing, advanced purification, and LNP design for targeted delivery.
Featured Speakers:
- Rok Sekirnik: Head of Process Development for mRNA and pDNA, Sartorius BIA Separations
- Dan Peer: Professor and Director of the Laboratory, Precision NanoMedicine
Speaker: Rok Sekirnik, Head of Process Development for mRNA and pDNA, Sartorius BIA Separations
Abstract:
This presentation outlines a streamlined mRNA manufacturing approach, emphasizing high-quality DNA templates for RNA purity. It features a scalable purification workflow with continuous in-line lysis and selective hydrophobic interaction chromatography (SHIC) for supercoiled (sc) DNA purification. Efficient sc pDNA linearization is monitored by the PATfix system. In vitro transcription (IVT) is tracked with PATfix, monitoring nucleoside triphosphate (NTP) consumption. Using a DOE approach, a reaction yield of 25 g/L is achieved, transforming from batch to fed-batch mode. mRNA is purified with Oligo dT affinity chromatography, selective for polyadenylated mRNA, with residual dsRNA removed via reverse-phase or aqueous denaturing chromatography. The process scales from micrograms to multi-grams, suitable for clinical manufacturing.
Speaker: Dan Peer, Professor and Director of the Laboratory, Precision NanoMedicine
Abstract:
Modulating gene expression for tumor cell death, protein replacement therapy, or gene editing is a major medical challenge. Strategies have been developed to target cell surface receptors and create amino ionizable lipids for repeated dosing. Novel mRNA sequences with enhanced immunogenicity are designed for bacterial infections. Dan Peer will discuss the journey from Lipid Nanoparticles (LNPs) conceptualization to clinical application, including cancer targets, the first bacterial mRNA vaccine, and gene editing strategies. He will also cover targeting LNPs to the gut in inflammatory bowel diseases and the lung epithelium without targeting agents. Delivering RNA payloads to specific cells is a milestone in personalized medicine.
Session 2 | Mastering LNP Complexity: Manufacturing, Characterization, and Targeting
Explore the complexities of LNP development in our virtual event’s second session, where experts from NeoVac, NanoCell, and Sartorius BIA Separations shared practical solutions for overcoming CMC hurdles and enhancing therapeutic efficacy. This session delves into purification, analytical advances, and targeted delivery.
Featured Speakers:
- Heinrich Haas: Chief Technology Officer, NeoVac
- Tristan Kovačič: Project Manager Process Analytics Development, Sartorius BIA Separations
- Mojca Tajnik Sbaizero: Process Development Manager, Sartorius BIA Separations
- Jacek Lubelski: Chief Technology Officer, NanoCell Therapeutics
Speaker: Heinrich Haas, Chief Technology Officer, NeoVac
Abstract:
Following their use in Covid-19 vaccines, lipid nanoparticles (LNPs) with mRNA are gaining attention for vaccines against infectious diseases and siRNA delivery for rare diseases. Despite interest, critical quality attributes of LNPs, especially supramolecular organization, remain poorly understood. We present recent findings on advanced characterization of LNP size and structure based on their environment, offering insights for quality evaluation and understanding biological processes like endosomal release, potentially guiding future mRNA delivery systems.
Speaker: Tristan Kovačič, Project Manager Process Analytics Development, Sartorius BIA Separations
Abstract:
Lipid nanoparticles (LNPs) are key for nucleic acid delivery, with rapid scale-up during COVID-19. Their complexity poses challenges in quality and safety control, especially with targeted LNPs. Monolithic columns provide a solution for intact chromatography, addressing CMC challenges with advanced platforms using multiple analytical columns and detectors. This method separates cargos in formulations for CAR-T, CRISPR, and vaccines, determining nucleic acid content, particle heterogeneity, and purity, enhancing therapeutic efficacy and accelerating LNP-based treatments.
Speaker: Mojca Tajnik Sbaizero, Process Development Manager, Sartorius BIA Separations
Abstract:
Lipid nanoparticles (LNPs) are crucial for nucleic acid delivery, advancing therapeutic applications. Challenges in manufacturing and characterization include ensuring drug integrity and achieving efficient purification. CIM monolithic columns offer precise separation and uniform products, essential as complexity grows with multiple payloads or targeting moieties. Innovative analytics like PATfix LNP Switcher aid efficient processes. Monolithic columns provide superior purification compared to TFF and dialysis, optimizing recovery and functionality, ensuring consistent quality and efficiency in LNP-based therapies.
Speaker: Jacek Lubelski, Chief Technology Officer, NanoCell Therapeutics
Abstract:
The development of Chimeric Antigen Receptors (CARs) has revolutionized cancer therapy, but ex vivo CAR-T treatments face high costs, complex manufacturing, and logistical challenges. We introduce a cell-targeted lipid nanoparticle (tLNP) platform for in situ CAR-T cell generation, delivering DNA and RNA directly to T cells. This system activates primary T cells and enables stable CAR expression without traditional ex vivo steps. Our data shows successful CAR-T generation in vivo, offering scalable, cost-effective, and accessible CAR-T treatments, addressing key cell therapy challenges.
