Streamline Targeted LNP Development to Improve Preclinical Translation

Lipid nanoparticles (LNPs) have emerged as a leading platform for nucleic acid delivery; however, their inherent tendency to accumulate in the liver continues to limit broader therapeutic applications. Advances in lipid design, surface engineering and ligand conjugation are now enabling the development of targeted LNPs with improved specificity, reduced off-target effects and expanded potential beyond hepatic delivery.

This webinar will explore the formulation design and development of ligand-conjugated LNPs, with a focus on strategies for liver de-targeting and extrahepatic delivery. The featured speakers will highlight how advanced lipid systems and compositions can be leveraged to modulate protein corona interactions and reduce ApoE-mediated uptake, enabling more favorable biodistribution for the selective uptake by target cell types.

Panelists will also address the importance of iterative formulation optimization experiments to carefully assess and establish quality attributes for functionality and manufacturing feasibility. Approaches such as covalent conjugation and post-insertion methods will be discussed as strategies to enable controlled ligand incorporation while maintaining nanoparticle stability and performance. These considerations are critical to optimizing ligand density, orientation and overall target product profile performance suitability.

Furthermore, the session will examine in vitro models to evaluate targeted LNP activity and target engagement, including receptor binding, uptake and the potential for functional delivery in vivo, providing actionable data to guide formulation optimization.

Finally, the panel will explore the functional evaluation of targeted LNPs in preclinical models, focusing on the relationship between biodistribution, target expression and immunogenicity. Integrating these datasets is essential for understanding in vivo performance and nominating a candidate with empirically derived clinical potential.

Together, this discussion will provide a practical framework for advancing ligand-conjugated LNPs from formulation through preclinical validation, enabling more precise and effective targeted delivery strategies.

Register for this webinar to learn how LNPs can be optimized for targeted extrahepatic delivery and preclinical validation.

Speakers

(Moderator) Allen Horhota, PhD, Vice President, Platform and Delivery, Seamless Therapeutics

Allen Horhota, PhD, is an industry veteran in nucleic acid therapeutics and nanoparticle delivery, with nearly twenty years of experience advancing RNA medicines and delivery technologies. Trained as a Nucleic Acid Chemist and Enzymologist, Dr. Horhota earned his PhD in Chemistry from Boston College and completed postdoctoral training at The Scripps Research Institute, where he specialized in simplified Nucleic Acid Backbones, Chemical Synthesis and Enzymatic Processing.

Dr. Horhota is an Author and Inventor on multiple publications and patents spanning nucleic acid chemistry, nanoparticle targeting, polymeric delivery systems and lipid nanoparticle (LNP) technologies for RNA therapeutics.

Most recently, Dr. Horhota served as the Vice President of Platform at Seamless Therapeutics and Orna Therapeutics, where he was an early team member helping advance circular RNA and in vivo CAR-T technologies. Prior to Orna, he was an Associate Director in Moderna’s Delivery Innovation group, leading efforts in novel materials and process development for polymeric and lipid nanoparticle-based mRNA delivery systems. Earlier in his career, Dr. Horhota held scientific and leadership roles at BIND Therapeutics, where he focused on active nanoparticle targeting strategies and nanoparticle processing technologies for small-molecule and nucleic-acid payloads. Throughout his career, Dr. Horhota has contributed to multiple clinical and commercial-stage nanoparticle programs across Orna, Moderna and BIND Therapeutics.

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Nicholas Boylan, PhD, Senior Director, Scientific Services, Phosphorex

Nicholas Boylan leads a team of Scientists and Engineers specializing in the development and optimization of novel drug delivery systems, including lipid nanoparticles (LNPs) and polymeric nanoparticles.

Before joining Phosphorex, Nick directed analytical and formulation development of a novel soft-mist pulmonary inhaler at Molecular Infusions and contributed to the encapsulation of a wide range of payloads within the proprietary nanomedicine platform ACCURINS(R) at BIND Therapeutics.

Nick holds a Bachelor’s Degree in Chemical and Biomolecular Engineering from Cornell University and a Doctor of Philosophy in Chemical and Biomolecular Engineering from Johns Hopkins University.

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Syed Reza, MD-PhD, Licensing and External Innovation Alliance Management, NOF Corporation

Syed Reza brings twenty years of experience in biopharma and specializes in nanomedicine technology. Syed currently leads the External Innovation and Alliance activities at NOF Corporation’s Drug Delivery Solutions business unit. Recently, Syed has consulted for various CDMOs on commercial strategy in the manufacturing of complex biologics, including for Octoplus B.V, a Dutch nanomedicines company.

At Octoplus, Syed was responsible for securing partnerships on platforms for protein microspheres and lipid nanoparticles. These efforts led to the successful acquisition of Octoplus by Dr Reddys Laboratories in 2012.

Prior to joining Ootoplus, Syed was part of the large-scale peptide manufacturing team at Hoffman LaRoche and led efforts to launch the CDMO business and acquire two Phase III manufacturing contracts.

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Michelle Bellerose, PhD, Study Director, Neosome Life Sciences

Michelle Bellerose, PhD, is a Study Director at NeoSome Life Sciences, where she leads preclinical study design and execution across a broad range of therapeutic areas. With a strong foundation in translational research, Michelle applies her scientific expertise to guide complex research programs and generate high-quality data to inform critical development decisions from early discovery through proof-of-concept.

Prior to joining NeoSome, Michelle held research roles in academic, government and biotechnology organizations, including at Moderna, where she contributed to vaccine development programs. Her experience spans experimental design, preclinical assay development, cross-functional collaboration and scientific program leadership.

Michelle earned her PhD in Molecular Genetics and Microbiology from the University of Massachusetts Medical School and has more than a decade of experience spanning diverse research environments. She is passionate about applying rigorous scientific approaches to accelerate therapeutic development and help innovators navigate the challenges of early-stage research and preclinical evaluation.

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