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.

Christopher McKinney; Hemalatha G. Rangarajan; Leyla Shune; Ashley Gray; Haydar Frangoul; Jonathan D. Fish; Claudia Pagan; Sonali Chaudhury

doi:10.1016/j.jtct.2025.12.038

February 1, 2026Transplantation and Cellular Therapy

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.

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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.

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Implication

Observational analysis reveals the development of gene therapy cell products for sickle cell disease and thalassemia, indicating viable treatment feasibility.

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McKinney et al. (2026) studied this question.

synapsesocial.com/papers/69a760b9c6e9836116a2dc13 https://doi.org/10.1016/j.jtct.2025.12.038

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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

No abstract yet

Implication

Discussion

Cite This Study

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

No abstract yet

Implication

Discussion

Cite This Study

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