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Presentations
Overview of CBER’s Manufacturers Assistance and Technical Training Branch (MATT)
Loni Warren Henderson
Public Affairs Specialist
Manufacturers Assistance and Technical Training Branch
Division of Manufacturers Assistance and Training
Office of Communication Outreach and Development
Center for Biologics Evaluation and Research (CBER)
US Food and Drug Administration (FDA)
Regulatory Resources and Avenues for Obtaining Early Guidance from CBER/OTP
Heather Erdman, MCPM, RAC, CQPA
Associate Director of Quality Assurance
Office of Review Management and Regulatory Review (ORMRR)
Office of Therapeutic Products (OTP)
Center for Biologics Evaluation and Research (CBER)
US Food and Drug Administration (FDA)
CBER’s CMC Considerations for Early Phase Studies of Cell and Gene Therapy Products
Karin Knudson, PhD
CMC Reviewer
Office of Cellular Therapy and Human Tissue CMC (OCTHT)
Office of Therapeutic Products (OTP)
Center for Biologics Evaluation and Research (CBER)
US Food and Drug Administration (FDA)
Nonclinical Assessment of Cell and Gene Therapy Products to Support an IND
Devaveena Dey, PhD
Pharmacology-Toxicology Reviewer
Office of Pharmacology-Toxicology (OPT)
Office of Therapeutic Products (OTP)
Center for Biologics Evaluation and Research (CBER)
US Food and Drug Administration (FDA)
Clinical Consideration for Cell and Gene Therapy in Early Phase Study
Jessica Lee, MD, PhD
Branch Chief for the Oncology Branch 2 (OB2)
Division of Clinical Evaluation Oncology (DCEO)
Office of Clinical Evaluation (OCE)
Center for Biologics Evaluation and Research (CBER)
US Food and Drug Administration (FDA)
Abstract
This webinar offers an insightful overview of the journey of developing biological products, from concept through clinical trials. It begins by introducing the CBER Manufacturers Assistance and Technical Training Branch (MATTB), detailing its mission to provide timely, accurate, and useful information to stakeholders, primarily through responding to industry inquiries and coordinating liaison meetings. The presentation then explores the CBER Office of Therapeutic Products (OTP), highlighting critical regulatory resources and avenues for early guidance, including formal meetings like INTERACT and pre-IND meetings, alongside various online learning tools. A significant segment addresses Chemistry, Manufacturing, and Controls (CMC) considerations for early phase cell and gene therapy products, emphasizing the unique manufacturing challenges, the paramount importance of designing safety and quality into the product early, and strategies to avoid common issues in IND submissions. Furthermore, the discussion covers essential non-clinical assessment considerations, outlining regulatory mandates for toxicology studies, the selection of appropriate animal models, and the benefits of early engagement with the FDA for non-clinical program development. Lastly, the presentation delves into key clinical considerations for oncology trials, focusing on study design, endpoints, dose selection and escalation, and robust safety monitoring plans, underscoring that each complex cell and gene therapy requires individual evaluation due to its distinct characteristics and inherent safety concerns.