As a class of therapeutics, cell and gene therapies are remarkably diverse in their modes of action and composition. But perhaps even more remarkable is how the diversity of these modalities continues to expand, powered by transformational scientific advancements. Over the past few years, we have witnessed how induced pluripotent stem cells, gene replacement therapy, and genetic editing of various different cell types (T-cells, NK cells, stem cells, etc.) have made the transition from research tools to become the basis of clinical candidates in development. Cutting edge analytical methodologies which once were firmly in the sphere of discovery biology have been adopted into product development workflows. Personalized medicine approaches, such as the identification and targeting of tumor-specific neoantigens, are quickly evolving. Strategies are under constant development to better understand the regulatory implications of these new technologies.
In this session, we will explore examples of how new technologies have emerged in application to potential therapeutic products and discuss the practical CMC challenges that face those who develop and regulate them.
1. What are the CMC and regulatory factors that need to be considered when developing a pluripotent stem cell-based therapy product? 2. Why is it important to implement state-of-the-art technologies and algorithms into neoantigen identification pipelines? 3. How are innovations implemented in a regulated clinical environment? 4. How do regulators view the integration of emerging technologies in cell and gene therapy products?