Welcome to Prime Therapeutics’ (Prime’s) inaugural Cell & Gene Pipeline Outlook! This publication tracks what lies ahead for the cell and gene therapy (CGT) pipeline over the next few years. The pipeline for cell therapies — including CAR T — and gene therapies is dynamic and poised for growth as science and technology advances. 

Our team of experts has compiled updates regarding both novel experimental medicines and new uses or formulations for existing therapies, with several pending U.S. Food and Drug Administration (FDA) approvals. While the bulk of the agents profiled are for rare or orphan conditions, therapies for more common conditions are also featured. Each listing also includes product development status — whether an FDA application has been submitted or if safety and efficacy are being studied in Phase 2 or 3 clinical trials. 

These regenerative treatments can take different forms but a few principles apply. These products use cells and genetic material to treat or prevent disease. Although a number of them require multiple doses, many hold the promise to cure intractable diseases with just a single dose. These are highly complex, high-cost therapies, and they are not without risk. Moreover, their durability of response remains to be determined. Still, CGT has the potential to alter the trajectory of certain diseases and bring hope to patients and their families living with incurable or debilitating conditions.   

We hope you find this report insightful!