Assessing Developmental, Reproductive and Germline Transmission Risks for Cell and Gene Therapies

Gene and cell therapies, including AAV-mediated gene transfer, gene editing, and LNP-mRNA platforms, are transforming medicine while raising complex questions around developmental and reproductive toxicity (DART) and germline transmission risk assessment. The Developmental and Reproductive Toxicology (DART) and Cell and Gene Therapy – TRacking, Circulation and Safety (CGT-TRACS) Committees are co-organizing a two-day workshop that will convene experts from industry, academia, and global regulatory agencies to share best practices, review case studies and emerging tools, and identify gaps and harmonization opportunities.

Workshop sessions will include:
– Foundations of developmental, reproductive and germline transmission risk in cell and gene therapies
– From PCR Signal to Risk Assessment: Interpreting Gonadal Biodistribution and Defining Next Steps
– Developmental and Reproductive Toxicology Considerations
– Juvenile and Pediatric Considerations

The workshop will take place September 29-30 in Washington, DC, with a virtual participation option available. Spaces for both in-person and online participation will be limited. Registration will open later this spring/early summer. For more information or questions, contact Dr. Connie Chen (cchen@hesiglobal.org).