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Report from the FDA CBER Office of Therapeutic Products (OTP) Advanced Manufacturing and Analytical Technologies (AMAT) for Regenerative Medicine Therapies (RMT) Workshop


Ashley Krull, Ph.D.
Associate Director, Cell Therapy Manufacturing and Engineering
James Cancer Hospital Cellular Therapy Laboratory 
Assistant Professor, Hematology
The Ohio State University
Columbus, Ohio, United States


On March 14th, 2023, the FDA held an engaging virtual workshop to facilitate interactions and knowledge-sharing between staff from the Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) and experts in advanced manufacturing and analytical technologies (AMAT) for regenerative medicine therapies (RMT). All stakeholders in cell and gene therapy were also invited to listen to the presentations and panel discussions. The expressed goals of the workshop were:

  • To obtain knowledge from experts in the field of AMAT and RMT
  • To discuss innovative manufacturing technologies and alternative testing methods
  • To share experiences, challenges, and best practices critical for chemistry, manufacturing, and controls (CMC) of cell and gene therapies and tissue-engineered medical products

The all-day workshop agenda featured an international group of experts from academia, government, and industry. The day was divided into two sections: the joint Cellular Therapy/Tissue Engineering Session and the Gene Therapy: Viral Vectors Session. The recordings from both sessions are now available on YouTube via the links above or on the workshop page.

The presenters and topics spotlighted in the Cellular Therapy Session (with timestamps to the individual presentations) were:

  • Keynote Speaker Krishnendu Roy, Ph.D. – “Enabling Quality-by-Design (QbD)-driven Manufacturing of Cell Therapies: The Role of Data Science, In/At-line Process Analytics, and Feedback-controlled Automation” [10:00]
  • Sai Kiang Lim, Ph.D. – “Manufacturing Mesenchymal Stromal Cell-small Extracellular Vesicles (MSC-sEVS) for Clinical Testing” [55:08]
  • Jane S. Lebkowski, Ph.D. – “Scaling Manufacture of Pluripotent Stem Cell-Based Therapies for Advanced Clinical Development and Eventual Commercialization: Case Study of Development of an Implant for the Treatment of Geographic Atrophy” [1:25:05]
  • Qasim Rafiq, Ph.D. – “Digital Tools and Adaptive Manufacturing Strategies for Advanced Therapies” [1:53:10]


The presenters and topics featured in the Tissue Engineering Session [2:20:30] were:

  • D. Kacy Cullen, Ph.D. – “Tissue Engineered Medical Products for Nervous System Reconstruction” [2:21:30]
  • Warren L. Grayson, Ph.D. – “3D-Printed Biodegradable Scaffolds for the Treatment of Critical-Sized Midfacial Bone Injuries” [2:47:30]
  • Grace Zhang, Ph.D. – “Advanced 3D/4D Bioprinting and Nanotechnology for Complex Tissue Regeneration” [3:12:30]

Following the first panel discussion, the workshop moved into the second main session (Gene Therapy: Viral Vectors). This session included the following speakers and presentations:

  • R. Jude Samulski, Ph.D. – “The ABC of AAV & The Impact of AAV Biology on Efficient Vector Production” [24:00; Note: the first 20 minutes of the recording were erroneously duplicated]
  • Steven Gray, Ph.D. – “Overcoming AAV CMC Challenges Through the Use of Platform-Based Approaches to Streamline AAV-Based Drug Development” [1:10:50]
  • David Schaffer, Ph.D. – “Directed Evolution of EnhaAAV Delivery Systems for Clinical Gene Therapy” [1:42:00]
  • Ali Aghajanirefah, Ph.D. – “Gene Therapy for Prevalent Diseases: Strategies to Improve AAV Titer and Quality” [2:04:30]

These dynamic presentations culminated in panel discussions featuring audience questions. ISCT members are highly encouraged to view the workshop recordings and connect with the presenters, many of whom will be attending the 2023 ISCT Annual Meeting in Paris.

1. Advanced Manufacturing and Analytical Technologies for Regenerative Me. (2023, April 6). U.S. Food And Drug Administration.



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