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Ying Huang & Jakob Reiser, FDA/CBER: “Safety Considerations for Gene Editin...

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Room N-114, Genentech Hall

600 16th Street

UCSF Mission Bay

San Francisco, CA 94158

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Join us to hear from two experts at the FDA's Center for Biologics Evaluation and Research (CBER). Dr. Ying Huang will discuss regulatory considerations for gene therapy products involving gene editing technologies. Dr. Jakob Reiser will describe ongoing work in his laboratory dealing with safety issues pertaining to lentiviral vectors.

Abstract

FDA/CBER has a long history of regulating gene therapy products, including genetically modified cellular products that are generated using integrating viral vectors. Development of HIV-1-based gene therapy vectors to deliver therapeutic genes into humans with various medical conditions has been ongoing over the past two decades. The risks associated with these promising vectors include activation of oncogenes during random integration of the vectors into the genome and the ability to spread from cell to cell through the formation of replication-competent lentivirus. This experience has contributed to CBER’s understanding in the regulation of products that incorporate use of gene editing (GE) technologies to generate investigational therapies intended to treat a disease.

The regulatory review of gene therapy products uses a science-based approach that considers the risks and benefits of each investigational product in the framework of the respective clinical trial. We will describe the safety concerns associated with gene therapy products, including those that incorporate GE technologies, in the context of our research efforts aimed to improve the safety profile of HIV-1-based lentiviral vectors.

The introduction of GE technologies to generate gene therapy products raises unique concerns regarding whether the GE component is appropriately designed and optimized for specificity, and how the gene editing process may impact the product safety profile. CBER’s considerations for the preclinical evaluation of gene therapy products involving GE technologies build upon the extensive scientific and regulatory history that exists for gene therapy products. Specific safety concerns include the potential for gene editing events at off-target sites, chromosomal translocations, and genomic instability. We will discuss CBER’s preclinical expectations for gene therapy products with a focus on the products that incorporate GE technologies.

About the Speakers

Ying Huang

Dr. Ying Huang has been a Pharm/Tox master reviewer at FDA/CBER/OTAT/DCEPT since 2004. She is responsible for the review of the preclinical evaluations for proof-of-concept and safety related to cell and gene therapies, and gene editing derived gene therapy products on oncology and non-oncology diseases. Her review activities have been involved in submissions of Investigational New Drug (IND) including pre-preIND and pre-IND, and Biologics License Application (BLA). In addition to her review work, she is one of the representatives for the US FDA in the International Pharmaceutical Regulators Forum (IPRF) / Gene Therapy Working Group (GTWG). She was the co-representative for FDA/CBER in the Interagency Coordinating Committee on the Validation of Alternative Methods (ICCVAM) during 2006 – April 2017. Prior to the FDA, Dr. Huang received her Ph.D. degree in Pharmacology and Toxicology at the University of Toronto, Canada, and subsequently an NIH IRTA fellowship at NIH/NIDDK before became a senior scientist at Genetic Therapy Inc., a Novartis Company.

Jakob Reiser

Dr. Jakob Reiser received a Ph.D. in Biochemistry from the University of Basel in Switzerland. He then did postdoctoral work in Virology and Protein Chemistry in the Department of Biochemistry, Stanford University School of Medicine as an American Cancer Society Junior Fellow and at the Imperial Cancer Research Fund Laboratories in London. At Stanford he helped develop the Western Blot technique and was instrumental in setting up the original chromatin immunoprecipitation method.

Dr. Reiser was a group leader and lecturer in Biotechnology at ETH (Swiss Federal Institute of Technology) in Zürich, Switzerland between 1986 and 1994. His work there focused on quorum sensing in Pseudomonads.

Dr. Reiser joined the NIH in Bethesda, MD in 1994 to gain experience in the emerging field of gene therapy. He worked with HIV-1-based lentiviral vectors since 1995. The group was among the first to develop such vectors.

Dr. Reiser moved to the Louisiana State University (LSU) Health Sciences Center in New Orleans in 1999 to join the Gene Therapy Program as Associate Professor of Medicine and as the Director of the Gene Therapy Vector Core. At LSU he gained experience applying such vectors in the context of in vitro, ex vivo and in vivo applications. Past in vivo applications included the delivery of therapeutic genes to the brains of mice afflicted with globoid cell leukodystrophy. This work was supported by R21 and R01 grants from the NIH on which he was a PI.

Dr. Reiser moved to FDA/CBER in 2008 as a PI. His laboratory at CBER uses genetic engineering methods to improve the safety of HIV-1-based lentiviral lentiviral vectors. He spends 50% of his time as a CMC reviewer of gene therapy files.

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Room N-114, Genentech Hall

600 16th Street

UCSF Mission Bay

San Francisco, CA 94158

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