bluebird bio, Inc. - BLUE STOCK NEWS

bluebird bio (Nasdaq: BLUE) will present a corporate update at the 41^st Annual J.P. Morgan Healthcare Conference on January 12, 2023, at 7:30 AM PT. CEO Andrew Obenshain will lead the session. Investors can access the live webcast on the bluebird bio website, where a replay will be available for 30 days. bluebird bio focuses on curative gene therapies for severe genetic diseases, including sickle cell disease and β-thalassemia, and is known for its industry-leading data in ex-vivo gene therapy.

bluebird bio, Inc. (Nasdaq: BLUE) has appointed Joseph Vittiglio as Chief Business and Legal Officer. Vittiglio, who has over 20 years of biopharmaceutical experience, previously held executive roles at Finch Therapeutics and AMAG Pharmaceuticals. His expertise spans licensing, mergers, FDA regulations, and quality operations. This appointment aims to strengthen the management team as bluebird bio transitions into a commercial-stage company focused on delivering gene therapies for severe genetic diseases.

bluebird bio announced that the FDA has lifted its partial clinical hold on lovotibeglogene autotemcel (lovo-cel) for patients under 18 with sickle cell disease (SCD). The company plans to resume enrollment and treatment of pediatric patients in Q1 2023, following a thorough investigation into previous anemia cases related to the treatment. bluebird is on track to submit a Biologics License Application for lovo-cel by the end of Q1 2023, having met FDA requirements and updated exclusion criteria for ongoing trials.

bluebird bio presented updated data on its gene therapies for beta-thalassemia and sickle cell disease at the ASH Annual Meeting. Key findings for ZYNTEGLO (beti-cel) include 90.2% of beta-thalassemia patients achieving transfusion independence, with long-term follow-up showing durable treatment effects. Lovo-cel's data revealed two anemia cases linked to alpha-thalassemia trait. A BLA for lovo-cel is anticipated by Q1 2023. Safety profiles for both therapies were consistent with known side effects, and ongoing studies continue to advance understanding of treatment safety and efficacy.

bluebird bio announced a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $102 million. The PRV was awarded due to FDA approvals of its gene therapies, ZYNTEGLO and SKYSONA, in 2022. CEO Andrew Obenshain stated that this non-dilutive capital strengthens the company's financial outlook, aiding the launches of these therapies and upcoming milestones, including a BLA submission for lovo-cel for sickle cell disease. The transaction, subject to closing conditions, marks a significant financial maneuver for the company.

bluebird bio reported third-quarter results highlighting the commercial launch of ZYNTEGLO and operational progress in gene therapies. The company ended Q3 with $186 million in cash and equivalents, while total revenue was $0.1 million, down from $1.0 million year-over-year. Research and development expenses decreased to $53.1 million, alongside a net loss of $76.5 million. bluebird is adjusting its Q4 financial guidance due to increased launch investments and post-marketing commitments. Chris Krawtschuk was appointed CFO, and the company anticipates significant milestones in 2023.

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.