ASGCT Holds Third Annual FDA Liaison Meeting

ASGCT held the third annual liaison meeting with the FDA’s Center for Biologics Evaluation and Research (CBER) on November 16. This year’s meeting was co-chaired by Adora Ndu, PharmD, J.D., chair of the ASGCT Regulatory Affairs Committee; and Robert Pietrusko, PharmD, a member of the ASGCT Regulatory Affairs Committee.

ASGCT members presented on three topics to staff of the Office of Tissues and Advanced Therapies (OTAT), providing ASGCT recommendations to the Agency on regulatory topics of mutual interest in the field. In addition, Wilson Bryan, M.D., director of OTAT, provided useful information on a variety of gene therapy development challenges, including disease model selection, preclinical study issues, endpoint selection, trial design, and patient experience data collection. 

View their presentation slides below. 

Brian Long, Ph.D., BioMarin 
Recommendations for immunogenicity testing requirements for AAV gene therapy 

Janet Benson, DVM, Lovelace Biomedical 
Recommendations for risk assessment requirements (safety and toxicology) for animal models and nonclinical data 

Wilson Bryan, M.D., OTAT 
Gene therapy development challenges: A regulatory perspective 

Jacob Elkins, M.D., SVP, Sarepta 
Innovative clinical study design for gene and cell therapies 

Read the summary of recommendations that ASGCT presenters provided to FDA.

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