Cell & Gene Therapies
By harnessing advanced science and novel technologies, cell and gene therapies have the potential to revolutionize cancer care. Friends of Cancer Research (Friends) works to build alignment in the cell and gene therapy development ecosystem to ensure these innovative therapies reach patients with serious and rare diseases. Friends draws on insights from emerging clinical programs and the U.S. Food and Drug Administration (FDA) approved cell therapies to identify scientific, operational, and regulatory challenges and to help advance the next generation of cell and gene therapies.
What is the unmet need and why does it matter?
Recent approvals and clinical experience with cell and gene therapies demonstrate their potential to improve outcomes for patients with cancer. Cell therapies, such as chimeric antigen receptor (CAR) T-Cell therapies, are a type of immunotherapy that aim to enhance or redirect a patient’s immune cells to attack cancer cells. Despite promising advancements with CAR-T therapies for treating hematologic malignancies and a tumor infiltrating lymphocytes (TILs) therapy for certain melanomas, there are several unique challenges throughout the drug development process that can slow progress. Current regulatory frameworks and development processes have the opportunity to evolve in parallel with rapid technological advances in cell therapies. Incorporating appropriate regulatory flexibility can help ensure that innovation continues without compromising patient safety.
How are we helping to find solutions?
Since the release of Friends’ first white paper related to cell and gene therapies in 2019, FDA published several guidance documents incorporating concepts we proposed, and FDA continues to highlight advances in cell and gene therapies as a priority area:
- Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial (Final Guidance, 2022)
- Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products (Draft Guidance, 2023)
- Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products (Final Guidance, 2024)
- Human Gene Therapy Products Incorporating Human Genome Editing (Final Guidance, 2024)
These guidance documents clarify regulatory expectations and outline areas where regulatory flexibility may be applied to support safe and efficient development of cell and gene therapy products. As the field gains additional experience and new challenges arise, Friends will continue to convene experts across sectors and propose regulatory policy solutions to advance the development and regulation of cell and gene therapy products.
How does this impact patients?
Cell and gene therapies offer the potential for substantial and durable benefit for patients with certain cancers. While scientific challenges remain in expanding their use more broadly, emerging technologies are helping overcome these challenges. Friends works to optimize development and policy that help ensure these therapies are safe, accessible, and able to reach more patients as the next generation of cell and gene therapies advances.
Project Outcomes
Annually, Friends hosts a public meeting focused on advancing cell and gene therapies:
2025: Unlocking Next-Generation Therapies
White Paper
Friends collaborated with the Parker Institute for Cancer Immunotherapy (PICI) to explore innovative development and manufacturing models for cell therapies, including a series of proposals focused on aligning regulatory flexibility, manufacturing adaptability, and sustainable development models that to expand the availability of genetically modified cell-based therapies for patients.
Discussions from this meeting were published in a peer review article entitled “Enabling Access to Genetically Modified Cell Therapies Through Flexible Approaches to Manufacturing and Cost Recovery” in JITC in 2025.
As a follow-up to the joint PICI meeting, Friends hosted a follow up webinar for advocates.
2024: Unlocking Complex Cell-based Gene Therapies
Publication
Friends collaborated with PICI and other experts to discuss innovative frameworks and proposals for enhancing the safety, expansion, and activity of the next generation of cell-based gene therapies.
Findings from discussions were compiled and published as a peer-reviewed article entitled “Intentional heterogeneity in autologous cell-based gene therapies: strategic considerations for first-in-human trials” in JITC in 2025.
As a follow-up to the joint PICI meeting, Friends hosted a follow up webinar for advocates.
2023: The Next Generation of Cellular Therapies: A Blueprint to Accelerate Development
The Next Generation of Cellular Therapies: Opportunities to Accelerate Development
Friends led multi-stakeholder discussions to evaluate how existing data can be safely leveraged across products and between versions of engineered cell therapy products to support more efficient development, iterative product improvements, and broader access to these therapies.
Findings from these discussions were published in a peer reviewed manuscript in 2024 entitled “Accelerating the development of genetically engineered cellular therapies: a framework for extrapolating data across related products” in Cytotherapy.
2021: Shaping the Future of Emerging Immunotherapies and Cell Therapies
Friends worked with leading experts in the field to describe the need for a standardized, consistent approach to defining and recording cytokine release syndrome (CRS) across trials — in order to improve accurate identification of CRS, facilitate risk benefit assessment, support better monitoring and management of toxicities, and ultimately improve patient care as immunotherapies expand.
This work culminated in a peer reviewed manuscript entitled “Need for aligning the definition and reporting of cytokine release syndrome (CRS) in immuno-oncology clinical trials” published in Cytotherapy in 2022.
2019: Designing the Future of Cell Therapies Meeting
Friends collaborated with PICI to identify potential approaches to address development and regulatory hurdles encountered during early- and late-stage development of cell and gene therapies.