Sickle Cell Anemia

Sickle cell anemia is a disease passed down through families in which red blood cells form an abnormal sickle or crescent shape. Red blood cells carry oxygen to the body and are normally shaped like a disc.

Programs + Services

Benign Hematology Program

The Hematology Program at Tufts Medical Center in Boston specializes in treating non-cancerous blood-related illnesses like bleeding and clotting disorders.
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Research + Clinical Trials

A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.

This is a non-randomized, open label, multi-site, single dose, Phase 3 study in approximately 35 adults and pediatric subjects > 2 and < 50 years of ages with SCD. The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using LentiGlobin BB305 Drug Product, an autologous CD34+ cell-enriched population that contains hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector (LVV) encoding beta A-T87Q-globin gene, suspended in cryopreservation solution in the final immediate container for the intended medical use.
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