FDA Lifts Partial Clinical Hold for bluebird bio’s Sickle Cell Disease (SCD) Studies for Patients Under the Age of 18

Company remains on track to submit for U.S. FDA approval of lovo-cel for SCD in the first quarter of 2023

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) lifted its partial clinical hold for patients under the age of 18 in studies evaluating lovotibeglogene autotemcel (lovo-cel) for sickle cell disease (SCD).

“We are very pleased to have addressed the FDA’s questions and resolved the partial clinical hold,” said Richard Colvin, M.D., Ph.D., chief medical officer, bluebird bio. “We are working closely with study investigators and clinical trial sites to resume enrollment and treatment of pediatric and adolescent patients in the first quarter of next year.”

In December 2021, lovo-cel clinical studies were placed on a partial hold for patients under the age of 18. The hold related to an investigation by bluebird bio into an adolescent patient with persistent, non-transfusion-dependent anemia following treatment with lovo-cel.

Results from a detailed investigation of this case were presented at the American Society of Hematology (ASH) Annual Meeting and Exposition on December 10, 2022, alongside details from another case of persistent anemia in an adult patient following treatment with lovo-cel. Both patients had two α-globin gene deletions (−α3.7/−α3.7), also known as alpha-thalassemia trait, and notably are the only patients in the study with this specific genotype. Following these cases, this genotype was added to exclusion criteria for ongoing studies.

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 U.S. FDA for lovo-cel in the first quarter of 2023.

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.

For more information, visit bluebirdbio.com or follow us on social media at @bluebirdbio, LinkedIn, Instagram and YouTube.

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 Securities and Exchange Commission. These risks include, but are not limited to: the risk that the Company may not realize expected cost savings from the restructuring, including the anticipated decrease in operational expenses, at the levels the Company expects; the Company may encounter additional delays in the development of its programs, including the imposition of new clinical holds or delays in resolving existing clinical holds, that may impact the Company’s ability to meet its expected timelines and increase its costs; the internal and external costs required for the Company’s ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected which may cause the Company to use cash more quickly than it expects or change or curtail some of its plans or both; the Company’s expectations as to expenses, cash usage and cash needs may prove not to be correct for other reasons such as changes in plans or actual events being different than the Company’s assumptions; the risk that the efficacy and safety results from the Company’s prior and ongoing clinical trials will not continue or be seen in additional patients treated with the Company’s product candidates; the risk that additional insertional oncogenic or other reportable events associated with lentiviral vector, drug product, or myeloablation will be discovered or reported over time; the risk that the Company’s eli-cel, beti-cel and lovo-cel programs may be subject to further delays in their development, including but not limited to the imposition of new clinical holds; the risk that lovo-cel may not be approved within the priority review timeframe or at all; and the risk that any one or more of the Company’s products and product candidates, including eli-cel, beti-cel or lovo-cel, will not be successfully developed, approved or commercialized. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, bluebird bio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

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