Gene therapy holds promise for treating countless diseases and disorders including cancer, cystic fibrosis, heart disease, diabetes, hemophilia, AIDS, and many more. Through use of a vector (viral or non-viral), a new copy of a gene or relevant nucleotide sequence is inserted into a patient’s cells ex vivo or in vivo to either compensate or correct for the deleted or defective version that is causing the disease, or enhance an existing version. The emergence of powerful new technologies such as the gene-editing tool CRISPR served as a breakthrough for the biotech industry and elicited a resurgence in the field, which is now being met by impressive research demand.
Currently, gene therapy is only usually available as part of a clinical trial, and product approvals using these technologies are just on the horizon with numerous technical challenges (and opportunities) associated with them. Pre-clinical and clinical research is ongoing to better understand how and when gene therapy will be effective in a patient. Particular needs include:
Improvements in vector safety
Regulation of gene expression
Enhanced cell transduction capability
Reference materials for viral tools
However, the manufacturing, and measurement and analytical techniques to support these processes are lacking.
The absence of a common set of standards in gene therapy programs has proven to be a significant challenge for regulators, industry leaders, and academic researchers, but there is no mistaking the utility, and, therefore, need, for standards (and related technologies) in these areas. Mike Kelly (Biogen) and Dr. Maritza McIntyre (Advanced Therapies Partners) Co-chair our effort to elucidate the specific needs for characterization and control in the gene therapy field and subsequently pioneer projects to bring us one step closer to establishing consensus standards.
Gene Therapy Chairs
Dr. Mike Kelly has more than 20 years of experience in the field of gene therapy. During this time, Mike has been responsible for various aspects of research, development and clinical manufacturing for a number of lentiviral, adenoviral and Adeno-associated virus (AAV) -based therapeutics. Currently, Mike works at Biogen, where he is a Director in the Asset Development and Portfolio Management group, leading the gene therapy portfolio. Prior to his current role, Mike was the Director of Gene Therapy Development at Genzyme Corporation, where he was responsible for process development and manufacturing for Genzyme's gene therapy programs. Prior to that, Mike was most recently the Site Director at Genzyme’s development and clinical manufacturing facility in San Diego. Before joining Genzyme, Mike worked at Cell Genesys and Somatix Therapy Corporation, where he held various roles in research and development and clinical manufacturing for gene therapy programs.
Dr. Maritza McIntyre has almost 20 years of experience in the development, evaluation and regulation of biological and small molecule products. She served as a reviewer and ultimately Branch Chief in the Division of Cellular and Gene Therapies at FDA/CBER, where she was actively involved in policy development and liaison activities to NIH, patient groups, ASCGT, and the AAV2 Reference Standard Working Group. She has since served as Director and Vice President of Regulatory affairs for Bavarian Nordic, REGENXBIO, Inc., and NanoCor Therapeutics, and as Executive Vice President of Regulatory Affairs and Product Development at Bamboo Therapeutics. Currently, she provides regulatory strategy and product development consulting to developers of advanced therapy products as an independent consultant at Advanced Therapies Partners, LLC. Dr. McIntyre received a Ph.D. in virology from the University of Chicago and graduated magna cum laude with an Honors B.S. in biology from Wayne State University.