AABB23: Unleashing the Future of CGTs through Standardization

It’s been just six years since the first chimeric antigen receptor (CAR) T-cell therapy received regulatory approval in the United States. Since then, the world has witnessed a tidal wave of innovation in cell and gene therapies (CGTs), with more than 2,000 clinical trials underway globally, 200 of which have advanced to Phase III.

However, as the number of CGTs continues to expand, so do the challenges faced by clinical centers that support their manufacture. These obstacles, along with potential solutions, took center stage during a Sunday morning Annual Meeting session.

Kicking off the session, Hien D. Liu, medical director of the apheresis program at Moffitt Cancer Center, provided an eye-opening view of the current landscape for clinical centers involved in CGT manufacturing. Liu laid out how several challenges tied to T cell harvesting by apheresis stem from variation. From agreements to electronic systems, forms to labeling – each step comes with its unique complexities. Liu wrapped up with insights into ongoing efforts for harmonization and partnerships. Government agencies, industry working groups, and standards-setting organizations are all working to navigate these challenges.

The session continued with a presentation by Ronit Slotky, PhD, MSc, director, Cell Therapies Manufacturing Facility, Hackensack University Medical Center. Slotky delved into the logistics and financial complications introduced by the varying manufacturing processes in CGT manufacturing. She also illustrated how variation can cause even seemingly simple tasks, like drug receipt, to become overwhelming. She also addressed challenges in drug preparation, communication, record-keeping, and oversight.

Slotky then outlined a roadmap for clinical centers to prepare for the future:

• Create a process for product onboarding.
• Develop product-specific batch records or checklists. These can help capture protocol-specific information, required documents and specific tasks.
• Create a budget for services and list the allocated resources for investigational products. This can help justify the department’s financial and operational needs.

Slotky underscored the urgency of action as more breakthrough therapies make their way into the pipeline. But where to begin? She offered several recommendations:

• Standardization of IT platforms.
• Recognition of established accreditations to ease the burden of onboarding.
• Standardizing approaches to agreements and contracts.
• Feedback reporting from manufacturers.
• Proper reimbursement for ancillary services.