FDA Expectations for Cell, Tissue and Gene Therapy Products

Cell, tissue and gene therapies represent an important and burgeoning class of FDA-regulated therapeutic products for treatment of disease ranging from AIDS to cancer. How does FDA view and regulate these products and what types of nonclinical studies are required to reach Phase 1 clinical trials and beyond? Getting FDA input at the earliest stages is crucial, and the pre PRE IND will be discussed as a venue for obtaining this early FDA feedback on your development program. Additionally, this talk will highlight the novel properties of these products, and what must be considered when designing nonclinical programs to support a US IND and ultimate licensure. Elements of study design will be discussed including study size, duration, choice of species and endpoints. The importance of GLP compliance, as well as preparing an FDA-ready IND package will also be presented.

Areas Covered in the seminar:

• Unique properties of Cell and Gene Therapy Products.
• FDA Regulation of cell and Gene therapy products.
• The pre PRE IND process and working with the FDA.
• Efficacy and proof of concept studies - starting out on the right foot!
• Key considerations for nonclinical safety studies.
• Biodistribution Studies - Where does your product go?
• Tumorigenicity Data - An important piece of the puzzle.
• Presenting nonclinical Data to the FDA in your IND or BLA.

Who will benefit:

This webinar will be valuable for anyone developing cell, tissue or gene therapy products, including those in the biopharmaceutical industry or looking to commercialize novel therapies out of an academic or research environment as well as those with experience in small molecule drugs who have little experience dealing with OCTGT Products. Those who might benefit include:

• Regulatory professional’s crossing over into cell and gene therapy products
• Researchers or academics wishing to commercialize cellular products
• Biotechnology Scientific staff
• CRO Study directors and technical staff
• Sr. Managers and CEOs of cell and gene therapy companies

Instructor Profile:

David Pepper, Ph.D., is a toxicologist with the Biologics Consulting Group, a consulting group specializing in the development and regulation of biologic products. David designs and develops nonclinical programs for small molecule and biologic products. He works closely with sponsors and the FDA to navigate regulatory paths for cell and gene therapy products, and his recent experience includes early stage preclinical development planning and nonclinical study design, regulatory toxicology, GLP toxicology study oversight and monitoring, as well as both pre-IND and IND review and preparation.

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