Tailoring Preclinical Design to LNP Chemistry
Recent advancements in combinatorial chemistry are yielding LNP platforms with highly specific biological properties. The development of biodegradable ionizable lipids, for instance, directly addresses historical limitations in therapeutic delivery by improving the safety and efficacy profile (PMID: 40060499). Similarly, the integration of novel chemical moieties into lipid backbones can augment intracellular processing and achieve organ-specific mRNA delivery, such as to the liver or lung (PMID: 39354147).
These molecular-level modifications have significant implications for preclinical study design. A program utilizing an engineered LNP for targeted lung delivery will require a different biodistribution and toxicology assessment than a program using a conventional LNP optimized for hepatic expression.
Our scientific teams design and execute in vivo rodent studies that account for these nuances. Key activities include:
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Dose Range Finding: Establishing exposure-response relationships in relevant rodent models.
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PK Profiling: Characterizing the concentration of the LNP and its payload in circulation and tissues over time.
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Non-Target Tissue Biodistribution: Quantifying the distribution of the therapeutic to establish a comprehensive safety profile.
These non-clinical evaluations are conducted within our expansive preclinical facilities, providing the robust, reproducible data required to justify human dose selection and meet US FDA regulatory expectations. This work is foundational to our 100% successful IND rate since 2019, a track record established by our principal scientists prior to the formal launch of Franklin Biolabs in 2024.
This detailed characterization is a core component of the services available through our Preclinical | Translational Services group, ensuring your program advances with a clear understanding of its in vivo behavior.
