First Patient Receives Treatment in Amended Sickle Cell Study

bluebird bio announces first patient treated with LentiGlobin drug product under amended study protocol in HGB-206 Phase 1 study of patients with severe sickle cell disease.

bluebird bio, Inc., a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced treatment of the first patient under the amended study protocol in HGB-206, the company’s Phase 1 study of its LentiGlobin drug product in patients with severe sickle cell disease (SCD). This study now incorporates several changes to the study protocol with the goal of increasing production of therapeutic anti-sickling hemoglobin (HbAT87Q).

“Our early clinical experience with LentiGlobin drug product in sickle cell disease has given us a deeper understanding of the biology of this complex disease,” said David Davidson, M.D., chief medical officer. “This research has informed numerous changes we have implemented in the HGB-206 study protocol that, in addition to the introduction of the transduction enhancers into our manufacturing process, we hope will improve in vivo VCN and HbAT87Q expression. The impressive drug product VCN achieved in the first patient under this amended protocol highlights the success of the changes to our drug product manufacturing process, and we are hopeful that these modifications will improve patient outcomes.”

Changes to the study protocol for HGB-206 include increasing the percentage of transduced cells through manufacturing improvements, improving myeloablation (and subsequent engraftment) by increasing the target busulfan area under the curve, introducing a minimum period of regular blood transfusions prior to stem cell collection, improved cell processing and exploring an alternate hematopoietic stem cell (HSC) procurement method. To accommodate these changes to the protocol, the study enrollment has been expanded for a total enrollment of up to 29 patients.

About the HGB-206 Study

HGB-206 is an ongoing, open-label Phase 1 study designed to evaluate the safety and efficacy of LentiGlobin BB305 drug product in the treatment of subjects with SCD. The study is designed to enroll up to 29 subjects. Subjects will be followed to evaluate safety and efficacy, which will be measured based on changes in red cell function tests, hemolysis markers, and frequency of clinical events secondary to SCD (e.g., vaso-occlusive crises or acute chest syndrome events).

For more information on the HGB-206 Study, please visit https://www.clinicaltrials.gov using identifier NCT02140554.

About SCD

SCD is an inherited disease caused by a mutation in the beta-globin gene that results in sickle-shaped red blood cells. The disease is characterized by anemia, vaso-occlusive crisis, infections, stroke, overall poor quality of life and sometimes, early death.

Where adequate medical care is available, common treatments for patients with SCD largely revolve around management and prevention of acute sickling episodes. Chronic management may include hydroxyurea and, in certain cases, chronic transfusions. Given the limitations of these treatments, there is no effective long-term treatment. The only advanced treatment for SCD is allogeneic hematopoietic stem cell transplant (HSCT). Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft-versus-host disease and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT.

(Source: Business Wire)