Bioanalytical Development And Testing For LNPs

Characterizing the biodistribution profile of lipid nanoparticle (LNP) formulations following in vivo administration is a critical regulatory requirement for gene therapy products. These studies provide essential data on the distribution of the therapeutic components — whether it's the mRNA payload, the lipid constituents of the LNP delivery system, or the protein expressed from the mRNA — across target, non-target, and germline tissues. Such information supports both efficacy and safety assessments, enabling informed regulatory submissions.

Biodistribution studies may focus on different analytes depending on the therapeutic goals and regulatory strategy. For example, the quantification of non-endogenous lipids using LC-MS/MS provides valuable insight into the distribution and clearance of the delivery vehicle itself. In addition to LC-MS/MS, a range of orthogonal methods, such as RT-qPCR for nucleic acid detection and ELISA for protein expression analysis, can be employed to provide a comprehensive biodistribution profile.

Since 2015, Pace® Life Sciences has supported sponsors and contract research organizations (CROs) with bioanalytical method development and validated testing services in compliance with 21 CFR Part 58 (GLP). Our centralized bioanalytical testing capabilities not only ensure consistent, high-quality data but also offer sponsors greater flexibility in CRO selection. This strategic advantage can streamline development timelines, reduce costs, and enhance the overall efficiency of gene therapy programs.