Welcome to Prime Therapeutics’ (Prime’s) Cell & Gene Pipeline Outlook! This evidence-based publication explores 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 therapies and new uses or formulations for existing treatments, with several awaiting Food and Drug Administration (FDA) approval. While most of the agents profiled target rare or orphan conditions, therapies for more common conditions are also included. Each listing provides information regarding development status — whether an FDA application has been submitted, or safety and efficacy are currently being evaluated 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 intractable diseases. While some require multiple doses, many hold the promise of a cure in a single administration. These are highly complex, high-cost therapies, and they are not without risk. Moreover, their durability of response remains uncertain. Nonetheless, CGTs have the potential to alter the trajectory of certain diseases, bringing hope to patients and families living with incurable or debilitating conditions.    
 
We hope you find this report insightful!