A Tailored Preclinical Strategy for Novel AAV Capsids

A standardized preclinical toxicology template is insufficient for the structural and functional diversity of today’s advanced therapeutics and complex biologics. For novel AAV capsids, achieving a predictable timeline to IND submission requires a bespoke, data-driven strategy from the outset. Applying proven intelligence in IND-enabling toxicology is how our teams accelerate these next-generation therapies, consistently moving candidates to IND in 18-24 months.

Each novel or engineered AAV capsid presents a unique profile for:

• Tissue and cell-type tropism
• Potential for unintended biodistribution and non-target tissue transduction
• Pre-existing and treatment-emergent immunogenicity

These variables demand a preclinical plan that is specifically adapted to the vector’s characteristics and the target indication. A failure to account for this specificity introduces significant risk into the development program.

Our approach begins with the vector itself. Historical data shows that capsid selection directly influences therapeutic expression and efficacy, as demonstrated in evaluations of vectors like AAVhu37 for hemophilia A (PMID: 29890905). Even subtle operational parameters can affect vector pharmacokinetics and safety profiles, reinforcing the need for customized study designs over rigid protocols (PMID: 27933307). Crucially, this requires establishing the clinical development plan early to ensure the test article is delivered in preclinical studies in the exact same manner intended for the clinic. This strict alignment of formulation and administration is foundational to our 100% IND success rate, ensuring toxicology studies generate robust data that satisfy both US FDA and EMA/MHRA IMPD expectations from day one.

Selecting a preclinical model with an immune system that closely mirrors the human response is a key component of a well-designed program. The goal is to generate data that is as predictive as possible for human clinical outcomes, which requires careful consideration of the biological relevance of the selected models.

This level of scientific collaboration is what defines a successful program.

“My relationship with… Franklin Biolabs dates back to 2007. And it really has been one of collaboration… They’ve really been a scientific partner… the quality of the people has really led to the quality of their product.”

— Dr. Gerald S. Lipshutz

This perspective is central to how we operate: as an embedded scientific extension of your team.

A tailored preclinical strategy, built on a deep understanding of AAV biology and executed with operational precision, is the most direct path to a successful regulatory submission.