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Cell and gene therapies (CGTs) offer transformative promise for patients by offering the potential for curative treatments across monogenic diseases, hematologic malignancies and now movement into other more prevalent disorders, in each case where traditional treatments have often fallen short. Developing these complex therapies depends on a rigorous process and often faces scientific, regulatory, and operational complexity. Expansion of cell and gene therapies into broader therapeutic areas like neurological diseases layers further challenges associated with complex delivery routes, and development of relevant analytical and biomarker strategies.
This session bridges the complexity of nuanced science driving preclinical to clinical development approaches, and venture capital investment perspectives all within the current challenge-laden CGT landscape. Scientific experts will share insights into key considerations driving program advancements across technologies and platforms designed to support emerging therapeutic areas within cell and gene therapies. Neurologically-focused CGTs will be used as a focal point to discuss integrated testing and development solutions across the full continuum—from discovery through post-market monitoring.
Additionally, a leading venture capital voice will provide insights into broader investment trends in the CGT space, with a special focus on long-term funding—a persistent challenge across the industry. The session will close with a forward-looking discussion on how strategic partnerships, data-driven development, and financial planning are essential to ensuring innovation continuity and patient impact.
Key Themes:
- Investment outlook and venture capital strategies for sustaining funding in the CGT ecosystem
- Unique testing and development needs for CGTs in emerging therapeutic areas
- Focus on CGT considerations during preclinical development and clinical trial testing for neurological diseases
- Role of a global CRO partner in supporting CGT pipelines across the product development continuum
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