RNA Leaders USA Congress

Democratizing RNA and DNA Therapeutic Development: Innovations in Delivery, Manufacturing, and Analytics

Date: September 10, 2025

Time: 11.15 am (mRNA track)

Speakers:

• Nejc Pavlin, Process Analytics Development Manager, Sartorius BIA Separations
• Ian Villamagna, Sales Specialist DNA RNA, Sartorius

Abstract:

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The rapid advancement of mRNA and DNA therapeutics has been catalyzed by the success of mRNA-LNP (lipid nanoparticle) platforms, however broad deployment remains constrained by complex and costly manufacturing, delivery inefficiencies, and limited analytical frameworks. Here, we present a comprehensive strategy to lower these barriers through a suite of next-generation technologies designed to accelerate, simplify, and improve the production and functional assessment of RNA and DNA Therapeutics.

First, we will highlight emerging technologies that can be built into delivery systems that transcend conventional LNP structures. We focus on novel cationic lipid architectures and rationally designed LNP formulations, or ready to use reagents, tailored for specific cell and tissue types. These innovations enable more precise targeting and improved biodistribution, thereby enhancing therapeutic index. We further explore advances in LNP manufacturing, including novel microfluidic-based platforms and high-throughput screening methodologies that offer scalability, reproducibility, and reduced cost. These technologies support rapid iteration cycles and empower both early discovery and clinical translation across multiple scales of production.

Post-formulation, LNPs require downstream processing to make the formulation applicable for in vivo applications. CIM® technology is uniquely suitable for LNP purification due to the low shear stress from laminar flow. Our innovative chromatographic purification process using CIM® OH columns for RNA-LNP products ensures high recovery and stability, effectively removing ethanol and unencapsulated RNA.

Finally, we introduce an innovative analytical platform, the PATfix LNP Switcher, designed for the comprehensive characterization of complex LNP-based biopharmaceuticals. It assesses critical quality attributes, including encapsulation efficiency, integrity, detection of nucleic acid-lipid adducts, and identification and quantification of lipid components, including those in combined medications, CAR-T applications, and CRISPR gene editing.

Together, these combined improvements represent a paradigm shift in how mRNA and DNA-LNPs are designed, manufactured, and validated – paving the way for broader access, more streamlined and scalable development, and more effective next-generation therapies.

About Conference: 

RNA Leaders combines the science & business of RNA Therapeutics & Vaccines. The community is focused on championing innovation, sharing new data, case studies & building the partnerships needed to bring discoveries to market.

Location: Boston, MA