bluebird bio Sells Second Priority Review Voucher for $95 Million

– Non-dilutive capital further strengthens Company’s financial position –

bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today announced that it has entered into a definitive agreement to sell its second Rare Pediatric Disease Priority Review Voucher (PRV) for $95 million.

bluebird bio was granted two PRVs upon the US Food and Drug Administration (FDA) approvals of ZYNTEGLO® (betibeglogene autotemcel) for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood cell transfusions and SKYSONA® (elivaldogene autotemcel) for the treatment of early, active cerebral adrenoleukodystrophy, on August 17, 2022 and September 16, 2022 respectively. On December 29, 2022, bluebird closed the sale of its first PRV for $102 million.

“The sale of our second priority review voucher further bolsters our financial position and provides an important source of non-dilutive funding for the company,” said Chris Krawtschuk, chief financial officer, bluebird bio. “Strengthening the company’s balance sheet – as we have continued to do through demonstrated fiscal discipline and the sale of both PRVs – ensures we enter 2023 with significant momentum behind the commercial launches of our two FDA-approved gene therapies, and the opportunity ahead in sickle cell disease.”

bluebird received a payment of $95 million upon closing of the transaction, which occurred simultaneously with the parties entering into the agreement. Jefferies LLC acted as the exclusive financial advisor to bluebird for this transaction.

About the Rare Pediatric Disease Priority Review Voucher Program

The Rare Pediatric Disease Priority Review Voucher Program is intended to encourage development of new drug and biological products for the prevention and treatment of certain rare pediatric diseases. Under this program, upon approval, the US Food and Drug Administration awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The voucher can be redeemed to receive priority review of a subsequent marketing application for a different product. PRVs may be sold or transferred, and there is no limit on the number of times a PRV can be transferred.

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, ZYNTEGLO and SKYSONA are registered trademarks of bluebird bio, Inc. All rights reserved.

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 Company’s prospects, opportunities in sickle cell disease, and financial condition and results of operations. 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 its 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 its 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 its 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 its 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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