Clinical Trials

Clinical trials of gene and cell therapies, from CAR-T and AAV gene replacement to CRISPR editing. Each trial links its registry record and results publications. It adds a curated verdict on the primary endpoint, key efficacy and safety findings, and the design lessons it holds for the next therapy: target, modality, delivery, dose and toxicity.

Name
Study Evaluating the Safety and Efficacy of KTE-C19 in Adult Participants With Refractory Aggressive Non-Hodgkin Lymphoma
Study of Efficacy and Safety of CTL019 in Pediatric ALL Patients
Efficacy and Safety Study of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)