Session V – Case Studies from Global CGTP Submissions

This session will cover cell and gene therapy products in late stage of development and approved by the FDA and/or EMA to demonstrate the main CMC challenges, the regulatory framework, and the overall experience with the Health Authorities to bring these medicines to patients. The innovative approaches and the regulatory outcome will be discussed in case studies to cover different approaches on development, main differences observed between EU and US, and lessons learned.

Dr. Fuller will present on the use of correlative analysis to develop specifications for idecabtagene vicleucel (Abecma®), an FDA approved Bristol Myers Squibb cell therapy to target BCMA, for the treatment of multiple myeloma. His talk will focus on the outcome of correlative analysis and the regulatory feedback on specifications.

Dr. Wagmare will present bluebird bio’s Marketing Authorization Application (MAA) journey for elivaldogene autotemcel (eli-cel, Lenti-D™), an investigational gene therapy for the treatment of patients with Cerebral Adrenoleukodystrophy, a rare, X-linked, metabolic disorder primarily affecting boys between the ages of 3 and 12 years. His talk will focus on potency assay development, process performance qualification strategy development, and regulatory procedures.

Dr. McQueen will present on storage and shipping conditions at ultra-low temperatures and the analytical complexity in support of CMC changes in the post-approval phase. She will present on the FDA and EMA approved IMLYGIC™ (talimogene laherparepvec), a genetically modified oncolytic viral therapy indicated for the local treatment melanoma recurrent after initial surgery.