Considerations for Cell and Gene Therapy Programs Entering the Clinical Space.

Cell and Gene Therapy (CGT) describes a broad category of medicinal products with potential applications to prevent and treat human disease in multiple therapeutic areas. These therapies leverage the use of modified nucleic acids, altered cells or tissue, or both. The modality, mechanism, route of administration, and therapeutic indication for a CGT product will influence the challenges and opportunities for early clinical development, some of which may be highly specific to the product under consideration. Both EMA and FDA encourage early interaction between Sponsor and Health Authority (HA) to align on key elements of the CGT development program.