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Symbol BLUE
Name bluebird bio, Inc.
Currency USD
Sector Health Care
IndustryGroup Pharmaceuticals, Biotechnology & Life Sciences
Industry Biotechnology
Market NASDAQ Global Select
Country United States
State MA
City Cambridge
Zipcode 2142
Website http://www.bluebirdbio.com
CRISPR Therapeutics has faced a challenging market, with shares down 23% year-to-date despite recent regulatory approvals for its gene-editing medicine, Casgevy, which treats sickle cell disease and beta-thalassemia. The Biden administration's new Medicaid initiative aims to improve access to Casgevy, potentially expanding its market significantly. With a projected peak annual sales exceeding $2.2 billion, the company is well-positioned for future growth, although it currently reports minimal revenue.
Editas Medicine will lay off 180 employees, about two-thirds of its workforce, as it shifts focus to “in vivo” gene editing after failing to find a partner for its sickle cell therapy. The company is ending development of its lead CRISPR medicine and will share more preclinical data in early 2025.
AstraZeneca and Daiichi Sankyo announced new study results for their cancer drug datopotamab deruxtecan, showing a median survival of 15.6 months in patients with EGFR-mutated non-small cell lung cancer. Agenus plans to cut annual spending by 60% to focus on a late-stage cancer immunotherapy, while Amgen is investing $1 billion to expand its manufacturing in North Carolina. The U.S. Patent and Trademark Office has withdrawn a proposed rule aimed at reducing patent thickets, and Bluebird bio is executing a reverse stock split to comply with Nasdaq rules. Merus received FDA accelerated approval for zenocutuzumab, its first approved product.
The Centers for Medicare & Medicaid Services has negotiated agreements with Vertex Pharmaceuticals and bluebird bio to include their sickle cell disease treatments, Casgevy and Lyfgenia, in a new Cell and Gene Therapy Access Model. This voluntary model allows states in the Medicaid Drug Rebate Program to apply for funding based on the drugs' effectiveness in improving patient outcomes. With over 100,000 individuals affected by sickle cell disease in the U.S., the initiative aims to enhance access to innovative therapies while providing budget predictability for state Medicaid agencies.
Halozyme Therapeutics has made an unsolicited bid to acquire Evotec for 11 euros per share, totaling around 2 billion euros, aiming to enhance its strategic position in the drug industry. Bluebird bio has recorded its first revenue from its gene therapy Lyfgenia for sickle cell disease, while BeiGene has rebranded as BeOne Medicines to emphasize its oncology focus and distance itself from its Chinese roots. Adaptimmune plans to lay off a third of its workforce to save $300 million and focus on its cell therapy Tecelra, while Abeona Therapeutics awaits a decision from the FDA on its gene therapy pz-cel for a rare skin condition by April 2025.
New data reveal a cancer risk associated with Skysona, a gene therapy for cerebral adrenoleukodystrophy (CALD), with seven cases of blood malignancies reported among 67 treated boys. While the therapy shows promise, concerns about its safety persist, prompting careful consideration by physicians and families. Bluebird bio, the therapy's developer, emphasizes patient well-being and informed decision-making amid ongoing scrutiny.
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