Company remains on track to submit for
“We are very pleased to have addressed the FDA’s questions and resolved the partial clinical hold,” said
Results from a detailed investigation of this case were presented at the
Enrollment and dosing for adult patients 18 and older in the HGB-210 study continued as planned while the partial hold was ongoing for patients under the age of 18; the Company is working to resume enrollment and treatment of patients ages 2-17 consistent with the study protocol. bluebird remains on track to submit a Biologics License Application (BLA) to the
As previously communicated, the Company has completed treatment of all patients in HGB-206 Group C, which will form the primary basis for efficacy in its lovo-cel BLA submission and expects to complete vector and drug product analytical comparability studies for the lovo-cel BLA in the fourth quarter of 2022.
The FDA previously granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation for lovo-cel.
About lovotibeglogene autotemcel (lovo-cel)
lovotibeglogene autotemcel (lovo-cel) gene therapy is an investigational one-time treatment being studied for sickle cell disease (SCD), that is designed to add functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once patients have the βA-T87Q-globin gene, their red blood cells (RBCs) can produce anti-sickling hemoglobin (HbAT87Q) that decreases the proportion of HbS, with the goal of reducing sickled RBCs, hemolysis, and other complications. bluebird bio’s clinical development program for lovo-cel includes the completed Phase 1/2 HGB-205 and ongoing Phase 1/2 HGB-206 and Phase 3 HGB-210 studies. bluebird bio is also conducting a long-term safety and efficacy follow-up study (LTF-307) for people who have been treated with lovo-cel in bluebird bio sponsored clinical studies.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.
With a dedicated focus on severe genetic diseases, bluebird has industry-leading programs for sickle cell disease, β-thalassemia and cerebral adrenoleukodystrophy and is advancing research to apply new technologies to these and other diseases. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward.
Founded in 2010, bluebird has the largest and deepest ex-vivo gene therapy data set in the world—setting the standard for the industry. Today, bluebird continues to forge new paths, combining our real-world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds.
bluebird bio is a trademark of bluebird bio, Inc.
bluebird bio Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements, including the Company’s statements regarding the expected timing and submission of a BLA for lovo-cel, its expectations regarding the timing of resumed enrollment and treatment of pediatric and adolescent patients in its clinical studies evaluating lovo-cel, the timing of completion of vector and drug product analytical comparability for the potential lovo-cel BLA and management’s plans, expectations and strategy. Such forward-looking statements are based on historical performance and current expectations and projections about the Company’s future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond the Company’s control and could cause the Company’s future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird bio’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K, as updated by the Company’s subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the
Source: bluebird bio, Inc.