Considerations for Cell and Gene Therapy Programs Entering the Clinical Space

Abstract

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 the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) encourage early interaction between sponsor and health authority to align on key elements of the CGT development program.

Novel Therapeutic Modalities: The Future is Now

“…With regard to the role of health authorities, regulatory agencies encourage early interaction with sponsors to align on key elements of CGT development programs, because each therapy will have a specific risk–benefit profile depending upon its platform and mode of delivery, therapeutic indication, and demography of study participants. The comprehensive review by Walford and coworkers of current FDA and European Medicine Agency (EMA) guidelines relating to CGT entering into the clinic should therefore be a timely resource 20 …”

“…The comprehensive review by Walford and coworkers of current FDA and European Medicine Agency (EMA) guidelines relating to CGT entering into the clinic should therefore be a timely resource. 20 Targeted protein degradation (TPD) is an emerging therapeutic approach that potentially modulates proteins that are "undruggable" with conventional small molecules. 21 Since the first TPD molecule that harnesses the ubiquitinproteasome system to degrade a target protein was reported, several biotech and pharmaceutical entered the field and disclosed programs in preclinical and early clinical development.…”