Gene editing holds the promise to revolutionize medicine due to its potential to correct or disrupt target DNA sequences with a putative long-lasting effect. To realize the promise of these revolutionary medicines, appropriate quality control is needed to ensure targeting specificity to avoid unwanted off-target or on-target effects, such as translocation and integrations, and to address challenges related to formulation and delivery of a biologically active product.

In this session, we will discuss different technologies that lead to the improved specificity and control strategies to ensure product quality and safety. Examples of in vivo and ex vivo gene editing approaches that reached the clinic will be presented. Progress in innovative approaches, such as base editing, prime editing, and the emerging epigenetic editing technologies will be covered.

Session Speakers:

Regulatory Perspectives on Gene Therapies Incorporating Human Somatic Genome Editing

Zhaohui Ye, CBER, FDA

Analytical Challenges with Gene Regulation and Gene Editing In-vivo and Ex vivo Gene Therapies

Michael Moloney, Sangamo Therapeutics

Epigenetic Editing: New Frontier for Genetic Medicines

Raj Poudel, Tune Therapeutics

Additional Panelists:

Barbara Bonamassa, AIFA

Premanjali Lahiri, Graphite Bio

Robin Wesselschmidt

Appearances