bluebird bio, Inc. - BLUE STOCK NEWS

bluebird bio, Inc. (NASDAQ: BLUE) will present at the 31st Annual Credit Suisse Healthcare Conference on November 8 at 5:00 p.m. PT, held at the Terranea Resort in Rancho Palos Verdes, CA. Interested parties can access the live webcast on the company's website, with a replay available for 90 days post-event. bluebird bio focuses on curative gene therapies for severe genetic diseases, including sickle cell disease and β-thalassemia, backed by extensive data and a commitment to patient communities.

bluebird bio announced FDA Accelerated Approval of SKYSONA (elivaldogene autotemcel) for boys aged 4-17 with early active cerebral adrenoleukodystrophy (CALD), a severe neurodegenerative disease. This therapy aims to slow neurologic dysfunction progression. The clinical hold on its development has been lifted. The treatment costs $3.0M and is expected to be available by late 2022 in select treatment centers. SKYSONA showed promising clinical data with significant MFD-free survival rates, yet carries risks of serious adverse effects including hematologic malignancy.

bluebird bio (NASDAQ: BLUE) announced that Jason Cole, Chief Strategy and Financial Officer, will leave the company on October 14, 2022, to pursue new opportunities after 8.5 years. During his tenure, he played pivotal roles and helped stabilize the company’s financial position. Katherine Breedis will serve as interim CFO as an external search for a permanent replacement begins. The announcement highlights the significance of leadership transitions at bluebird as it aims to enhance its commercial stage operations and continue its focus on gene therapies.

bluebird bio, Inc. (NASDAQ: BLUE) announced its participation in upcoming investor conferences, including the Wells Fargo Healthcare Conference on September 8, 2022, and the Morgan Stanley 20th Annual Global Healthcare Conference on September 13, 2022. The presentations can be accessed via the Investors & Media section of their website. bluebird bio focuses on curative gene therapies for severe genetic diseases, maintaining a deep commitment to patient communities.

bluebird bio has received FDA approval for ZYNTEGLO® (betibeglogene autotemcel), the first gene therapy for beta-thalassemia patients requiring regular blood transfusions. Priced at $2.8M, ZYNTEGLO aims to eliminate the need for ongoing transfusions, potentially saving patients up to $6.4M in lifetime healthcare costs. An innovative outcomes-based contract allows for a single upfront payment with up to 80% reimbursement if transfusion independence is not achieved. ZYNTEGLO will be available through specialized Qualified Treatment Centers, supported by the 'my bluebird support' program for patients and families.

bluebird bio has received FDA approval for ZYNTEGLO (betibeglogene autotemcel), a one-time gene therapy for treating beta-thalassemia in patients needing regular red blood cell transfusions. This therapy aims to provide transfusion independence and improve hemoglobin levels. Clinical data from Phase 3 studies showed that 89% of evaluable patients achieved transfusion independence, with durable results lasting up to 4 years. ZYNTEGLO is expected to revolutionize treatment for this severe, genetic disease affecting approximately 1,300-1,500 patients in the U.S.

bluebird bio reported significant advancements in gene therapy during Q2 2022, including unanimous FDA advisory committee support for beti-cel and eli-cel. If approved, both therapies could launch in Q4 2022. The Company ended the quarter with approximately $218 million in cash equivalents. Total revenue increased to $1.5 million from $0.1 million YoY, while net loss narrowed to $100.1 million from $155.8 million. The BLA for lovo-cel is anticipated in Q1 2023. bluebird bio aims to resolve clinical holds and is exploring financing options, including equity offerings and monetizing potential FDA vouchers.

bluebird bio announced that the FDA's advisory committee voted 13-0, endorsing betibeglogene autotemcel (beti-cel) as a viable treatment option for beta-thalassemia patients requiring regular red blood cell transfusions. If approved, beti-cel would be the first curative gene therapy for this condition in the U.S. The FDA has set a decision goal date for the Biologics License Application on August 19, 2022. With significant clinical data backing beti-cel, 89% of patients in trials achieved transfusion independence. This is a crucial step toward addressing the unmet medical needs of beta-thalassemia patients.

bluebird bio announced positive recommendations from the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee for their gene therapy eli-cel, aimed at treating early active cerebral adrenoleukodystrophy (CALD) in children. The committee voted 15-0 in favor of the therapy's benefits outweighing the risks. The Biologics License Application (BLA) is under priority review, with a PDUFA goal date of September 16, 2022. If approved, eli-cel will be the first gene therapy for CALD, providing an alternative for over 70% of CALD patients lacking matched sibling donors.