Allogeneic CAR T therapies offer off-the-shelf access but present unique CMC challenges. This presentation will discuss donor selection, non-viral gene editing, and current limitations in isolation, expansion, and purification technologies. We will also address fill finish, dose control, cryopreservation, and distribution considerations needed to enable a scalable and compliant manufacturing strategy.

Caribou’s patented chRDNA (CRISPR hybrid RNA-DNA) technology enables superior specificity and precision that drives complex genome editing, including multiplex gene knockout and insertion, while maintaining genomic integrity. chRDNA genome-editing technology is used to armor allogeneic cell therapies with checkpoint disruption and immune cloaking to enhance anti-tumor activity. Allogeneic, or off-the-shelf, CAR T cell therapies have the potential to provide broad access and rapid treatment for patients with hematologic malignancies.

How can regulatory flexibility accelerate safe patient access to transformative cellular and gene therapies? This presentation will outline FDA’s flexible approach to CMC requirements for CGT products seeking BLAs. It will review three recent, interrelated guidance documents showing how FDA maintains rigorous standards while adapting expectations to meet the challenges of CGT development, fostering safe, effective novel therapies for patients with serious conditions.

Satellite Biosciences has developed a low-cost cell therapy designed to treat patients with severe liver disease. Our breakthrough technology expands primary hepatocytes sourced from donor liver tissue into a cryopreserved, off-the-shelf therapy for on-demand clinical use. To support commercial viability, Satellite has focused on driving down cost of goods by conducting multiple design-of-experiments (DOE) studies, establishing a scalable manufacturing process, and building centralized manufacturing capabilities. Satellite plans to initiate a Phase 1/2 clinical trial testing our lead hepatocyte therapy, SB-101, in pediatric patients with severe urea-cycle disorders later in 2026.

The role of cell processing labs has evolved since the approval of the first CAR T cell, Kymriah, in 2017. Here, we will discuss how our program supports investigator-initiated clinical trials and prepares these products for eventual commercialization, while also supporting stem-cell transplants and commercial gene-therapy products such as Kymriah, Lyfgenia, and Casgevy.

Chimeric antigen receptor (CAR) T cell therapy has transformed the treatment landscape for hematologic malignancies, yet widespread adoption remains constrained by complex, time-intensive, and resource-heavy manufacturing processes. This talk will provide an in-depth overview of process development strategies and recent advances that have enabled the clinical success of CAR T products while highlighting key bottlenecks that limit scalability, affordability, and global access.

Despite the growing interest in cell and gene therapies, their accessibility remains a challenge. Successful manufacturing programs typically require contracting third-party organizations associated with a prohibitive financial burden, which stifles successful transition to late-stage trials and commercialization. Moreover, the delivery of many cell therapies to patients continues to require highly specialized settings and personnel, adding to the logistical complexity of getting product to patient, posing risks to products, and adding to the cost of treatments. We discuss these challenges and share insights into solutions we've implemented successfully.

Cell therapies introduce unique drug product and delivery challenges that differ substantially from traditional biologics. This presentation will explore strategies to develop robust drug product formulations, optimize storage and handling, and enable reliable administration at the point of care.

There are unique challenges to processing and testing cell-containing products that cannot be terminally sterilized. FDA guidance and USP chapters related to visible particulates will be discussed. Lessons learned using a real-life CGT case study will be presented.