dispersion for infusion clinical trials

A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects with Severe Sickle Cell Disease, βS/βC Genotype

This study looks at a new gene therapy, exa-cel, for people with severe sickle cell disease. It aims to see if a single treatment can stop painful sickle cell crises for a year or more. Researchers will check if it's safe and how well it helps patients.