bluebird bio, Inc. - BLUE STOCK NEWS

bluebird bio (NASDAQ: BLUE) announced an extension of the FDA review periods for its gene therapies, betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel). The new PDUFA goal dates are August 19, 2022 and September 16, 2022, respectively, allowing the FDA to review additional clinical data submitted by bluebird. Notably, this extension does not relate to new safety issues. If approved, these therapies will be the first of their kind in the U.S. for severe genetic diseases. The company also addressed a partial clinical hold on another therapy, lovotibeglogene autotemcel (lovo-cel).

bluebird bio, Inc. (Nasdaq: BLUE) has announced key updates for the 40th Annual J.P. Morgan Healthcare Conference, focusing on its gene therapy programs. The company anticipates FDA reviews for betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel) in 2022, with potential launches in mid-2022. The company aims to complete validation lots for its third therapy, lovotibeglogene autotemcel (lovo-cel), amid ongoing clinical holds. bluebird bio projects a cash balance of approximately $442 million, expecting a cash burn of less than $400 million in 2022 and potential cash inflows of $150-200 million from priority review vouchers.

bluebird bio (Nasdaq: BLUE) announced participation in the 40th Annual J.P. Morgan Healthcare Conference on January 12 at 3:45 p.m. ET. Interested parties can access the live webcast on the company's website's 'Events & Presentations' page, with a replay available for 30 days afterwards. bluebird bio focuses on curative gene therapies for severe genetic diseases like sickle cell disease and β-thalassemia, supported by extensive clinical data since its founding in 2010.

bluebird bio (NASDAQ: BLUE) announced the FDA has placed its clinical program for lovo-cel gene therapy for sickle cell disease on partial hold for patients under 18. This action follows an investigation into a patient experiencing persistent anemia post-treatment, though no malignancy has been found. Adult patient enrollment continues in ongoing studies. The company plans to respond to the FDA's inquiries and evaluates the potential impact on its biologics license application submission timeline.

bluebird bio announced that the FDA has accepted the Biologics License Application (BLA) for its gene therapy, eli-cel, aimed at treating cerebral adrenoleukodystrophy (CALD) in children. If approved by the PDUFA date of June 17, 2022, eli-cel will be the first gene therapy for CALD, a rare neurodegenerative disease. The therapy has shown promising efficacy, with 90.6% of patients achieving primary endpoints in clinical trials. However, the clinical program is currently on hold due to reports of myelodysplastic syndrome linked to the treatment. The FDA granted eli-cel several designations, highlighting its potential in addressing serious medical needs.

bluebird bio has released updated results from its Phase 1/2 HGB-206 study of lovo-cel, a gene therapy for sickle cell disease, highlighting improved biologic and clinical outcomes. The data show stable production of anti-sickling hemoglobin and resolution of severe vaso-occlusive events (VOEs) for up to 36 months. Patient-reported outcomes indicate significant enhancements in health-related quality of life. With a median follow-up of 17.3 months, 35 patients achieved near-normal hemoglobin levels, with no severe VOEs reported in the Group C cohort.

bluebird bio (Nasdaq: BLUE) presented new long-term data on betibeglogene autotemcel (beti-cel) at the ASH Annual Meeting. The results show that patients with β-thalassemia achieved transfusion independence for up to seven years, with 89% of phase 3 patients experiencing normal hemoglobin levels. The therapy potentially eliminates the need for regular transfusions and stabilizes iron markers, with 59% of patients able to stop iron chelation. The FDA is currently reviewing a biologics license application for beti-cel, with a decision expected by May 20, 2022.

bluebird bio (Nasdaq: BLUE) announced that the FDA has accepted its Biologics License Application for betibeglogene autotemcel (beti-cel) for priority review. If approved, beti-cel will be the first one-time treatment targeting the genetic cause of β-thalassemia, benefiting patients dependent on regular red blood cell transfusions. The PDUFA goal date is May 20, 2022. The application is based on extensive clinical data, demonstrating potential for transfusion independence in 89% of patients.

AavantiBio has appointed Dr. Jenny Marlowe as Chief Scientific Officer. She brings nearly 15 years of experience in the biopharmaceutical sector, previously serving at Bluebird Bio (NASDAQ: BLUE) as VP of Preclinical & Translational Development. Dr. Marlowe will lead scientific research and preclinical development at AavantiBio, which focuses on gene therapies for rare diseases. The company has secured over $107 million in Series A financing and is advancing its gene therapy pipeline, including partnerships with leading organizations such as the University of Florida.