Preclinical Evaluation of Advanced Therapeutics

Our work in preclinical programs for advanced therapeutics and complex biologics is focused on a single objective: generating definitive data that minimizes clinical risk. Applying proven intelligence in IND-enabling toxicology, we design and execute studies that directly address the specific biological questions posed by your asset, ensuring a clear path toward regulatory submission.

Modality-Specific Preclinical Service Platforms

A standard preclinical template does not exist for next-generation therapies. We construct a tailored, data-driven strategy specific to the modality and target indication.

• Platform-Specific Administration: Expertise in systemic and targeted administration routes for viral vectors (AAV8, AAV9, engineered capsids), non-viral LNP delivery systems for RNA payloads (mRNA, siRNA), and cell-based therapies.
• IND-Enabling Toxicology: All range-finding and pivotal toxicology programs are conducted under non-GLP conditions or GLP conditions with rigorous quality assurance oversight (as appropriate), designed to meet FDA and other global regulatory expectations.
• Integrated Pathology: A full-service platform provides comprehensive histology, multiplex ISH, digital image analysis, and histopathology to evaluate biodistribution, transgene expression, and safety profiles.
• Gene Editing Evaluation: Deep experience with preclinical programs for a variety of nuclease platforms, including CRISPR-Cas9 and base editors, focused on assessing on-target efficacy and off-target safety.

Assessment of Long-Term Efficacy and Safety

Our experience includes evaluating the long-term durability and safety of an AAV-delivered gene editing platform, confirming stable target engagement in preclinical systems (PMID: 33609733). This focus on long-term biological outcomes is a key component of a robust data package.

Methodological Rigor and Adherence to the 3Rs

Executing these complex programs correctly the first time is a scientific and ethical necessity. Our approach to study design is built on this principle, adhering to the 3Rs (Replacement, Reduction, and Refinement). This rigor ensures that data is definitive and that animal use is minimized, reflecting a deep commitment to responsible research.