Phase 3 Clinical Trial using haematopoietic stem cells to treat patients with beta-thalassemia opens.
Bluebird Bio, a Cambridge, Massachusetts-based biotech, last week opened its enrolment of a Phase 3 trial of its LentiGlobin gene therapy. BlueBird’s Lentiglobin gene therapy involves the extraction of patient haematopoietic stem cells (HSC), which are then inserted with targeted gene sequences and re-infused back into the patient.
In the case of patients with beta-thalassemia, a rare blood disorder, Bluebird hopes to reduce the number of transfusions needed. The newly initiated study will aim for transfusion independence, or a 12-month transfusion free period after a transplant with modified HSC.
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