Cellular immunotherapies (e.g., CAR T cells) are primarily used as an autologous therapy to treat cancer. As such, these therapies are currently generated in small batches for each patient. To generate enough modified cells for a single treatment cells are expanded in volumes from 1 L to 10 L.
Careful planning is important for all early drug development programs, but it is particularly critical in rare diseases where study populations are limited and precedents for drug development are lacking. By proactively preparing for this meeting, sponsors can set themselves up for productive discussions, which may help in identifying areas of regulatory flexibility.
As gene transfer vehicles, lentiviruses exhibit many desirable properties, such as high transduction efficiencies, the ability to infect both dividing and nondividing cells, and stable integration into the host cell genome. These properties make them well-suited for in vivo and ex vivo gene and cell therapies.