Enabling IND Approval: Preclinical In Vivo Considerations for Novel Cell and Gene Therapies
Capabilities
Recorded: July 2022
Preclinical evaluation of cell and gene therapy (CGT) products in vitro and in vivo helps characterize product profile throughout development. The cell and gene therapy space is expanding significantly, with many candidate therapies advancing into clinical testing and many more progressing through preclinical pipelines. It is a critical time to ensure your preclinical road map is well thought through to position your product for success. As the number of early-stage candidate products continues to increase, so does the need to define effective preclinical development strategies that support clinical translation. Given the diverse and often multifunctional nature of these therapies, it is not possible to define a one-size-fits-all preclinical program for cell and gene therapy. Instead, preclinical studies need to be customized to support the feasibility of the proposed administration route and appropriate application procedure, address the candidate product’s specific therapeutic properties, and fully characterize any potential safety concerns. This webinar will highlight through case studies the benefit of experience and how a data driven science based approach using clinically relevant modalities and end points can support a successful IND submission.
Key Messages on IND-Enabling Approvals:
- The diversity and biological properties of GT products necessitate a flexible testing strategy
- Preclinical study designs for CGT should be product specific (no “one-size-fits-all” approach and supported by scientific rationale/data)
- The correct animal model has to be selected for assessment of bioactivity and safety which demonstrates a biological response similar to that expected in humans
- Study protocols should be reviewed and approved by an Institutional Animal Care and Use Committee (IACUC) before conducting studies and all study tasks should be performed in an AAALAC accredited facility in accordance with facility Standard Operating Procedures (SOPs) and consideration of the 3Rs
- The right end points have to be selected which can demonstrate efficacy and functional rescue with appropriate imaging modalities which enable visualization of anatomical and physiological changes
- Correctly designed POC and safety studies with clinically relevant endpoints/outcomes will help establish benefit: risk profile in the IND submission
- A qualified and experienced preclinical team with experience is key