Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206

β-globin gene transfer may reduce or eliminate complications in patients with sickle cell disease (SCD). LentiGlobin gene therapy (GT) comprises drug product (DP) made from autologous hematopoietic stem cells (HSCs) transduced with the BB305 lentiviral vector (LVV) encoding β-globin with an anti-s ickling T87Q substitution (HbAT87Q). The safety and efficacy of LentiGlobin GT in adults with SCD is being evaluated in a phase 1 study, HGB-206 (NCT02140554). Patients were initially treated with DP made from bone marrow harvested (BMH) HSCs (Group [Grp] A, fully enrolled), then from DP made from B MH HSCs but using a refined manufacturing process (Grp B, fully enrolled), and subsequently from plerixafor mobilized HSCs (Grp C, currently enrolling).

Source: Biology of Blood and Marrow Transplantation - January 31, 2019 Category: Hematology Authors: Markus Y. Mapara, John F. Tisdale, Julie Kanter, Janet L. Kwiatkowski, Lakshmanan Krishnamurti, Manfred Schmidt, Alexandra L. Miller, Francis J. Pierciey, Weiliang Shi, Jean-Antoine Ribeil, Mohammed Asmal, Alexis A. Thompson, Mark C. Walters Tags: 81 Source Type: research