Mapping the Clinical Development Trajectory of Cell and Gene Therapy Products.

ABSTRACT: While cell and gene therapies (CGTs) have emerged as promising modalities to treat conditions with limited therapeutic options, their unconventional development is fraught with uncertainty, rendering them high-risk assets for many pharmaceutical companies. Here, we assess the clinical development trajectories of CGT products by estimating probabilities of successful clinical trial phase transitions and the likelihood of achieving regulatory approval. We included all CGT products entering clinical development from 1993 to 2023 and intended for marketing in the United States, Europe, Japan, Canada, and Switzerland. Associations between product success and characteristics were investigated. In sub-analyses, we examined the clinical trajectories of two promising product types, chimeric antigen

SUBMITTER: Elsallab M 

PROVIDER: S-EPMC12447590 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature