Welcome to Prime Therapeutics’ (Prime) inaugural Cell & Gene Pipeline Outlook! This publication tracks what is ahead in 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 advance. 

Our team of experts has curated 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 includes 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 and are fluid, 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. Yet 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!