Extrapolating from Foundational Vector Biology
Our scientific leadership’s historical work in characterizing novel viral vectors provides the foundational intelligence for our approach to non-viral systems. Investigations into primate-derived AAVs established foundational principles of how vector structure influences in vivo biodistribution and transduction efficiency (PMID: 12192090, 15975006). This deep understanding of vector-host interactions informs how we design and execute PK studies for synthetic platforms, ensuring we anticipate and measure the parameters that truly matter for clinical success.
A tailored preclinical strategy for polymeric and hybrid vectors focuses on several key analytical endpoints.
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Systemic Exposure & Clearance: Quantifying the concentration of the vector in circulation over time to determine its half-life and clearance rate.
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Non-Target Tissue Biodistribution: Assessing accumulation in key organs such as the liver, spleen, kidneys, and lungs to identify potential off-target toxicities.
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Payload Integrity & Release: Developing assays to differentiate between the intact nanoparticle and the released therapeutic payload (e.g., RNA, gRNA) within tissues.
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Metabolite Profiling: Identifying how the body breaks down the vector components over time.
Executing these complex studies requires significant infrastructure. Our >100,000 sq ft facility provides the specialized laboratory and animal housing space necessary to conduct these programs entirely in-house.
This integrated approach supports an accelerated path to regulatory submission. The core scientific team at Franklin Biolabs, which formally launched in 2024, has maintained a 100% successful IND rate since 2019, consistently moving candidate therapies to the clinical stage in an 18-24 month timeline. This track record is built on designing preclinical programs that generate unambiguous data for global regulatory bodies, including the FDA, EMA, and Swissmedic.
