Collection and Manufacture of Autologous HSC Gene Therapy Cell Product for Patients with Sickle Cell Disease (SCD) and Transfusion Dependent Thalassemia (TDT): A Real World Gene Therapy (ReGenT) Consortium Report.

Key Points

• Findings demonstrate successful collection and manufacture of personalized gene therapy cell products for sickle cell disease and thalassemia.
• A total of 92% of patients achieved an adequate number of cells for gene therapy applications.
• Observational analysis across multiple centers focused on the processes involved in gene therapy cell product development.
• Highlights the need for further studies to establish long-term efficacy and safety in diverse patient populations.