Cell And Gene Therapy Development – Three Key Challenges In 2023

By Katie McCarthy

It took approximately 50 years to progress from the initial proof-of-concept of virus-mediated gene transfer by Rogers and Pfuderer to the approval of the first gene transfer products in humans. Similarly, nearly 30 years passed from the demonstration of the chimeric antigen receptor concept by Gross et al. to the approval of the first chimeric antigen receptor T cell (CAR-T) products.

However, in 2023, the journey from proof-of-concept to product approval is significantly expedited. The U.S. Food and Drug Administration (FDA) has witnessed a surge in cell and gene therapy products in development, as evidenced from the increasing Investigational New Drug (IND) applications. The FDA anticipates that by 2025, there will be 10 to 20 approved cell and gene therapy products annually, based on an assessment of the current pipeline and clinical success rates.

Considering the current landscape, here are three critical factors for cell and gene therapy developers to overcome obstacles and advance development.