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Business Upturn
01-05-2025
- Health
- Business Upturn
Gene Therapy Momentum Builds Among Hematologists in Sickle Cell Disease Management Despite Ongoing Access Barriers, According to Spherix Global Insights
EXTON, PA, May 01, 2025 (GLOBE NEWSWIRE) — Patients with sickle cell disease (SCD) continue to face severe complications, despite advances in treatment that have helped mitigate some risks. Increased rates of blood clots, stroke, and other complications still significantly reduce life expectancy and impact quality of life.1 SCD, a genetic disorder affecting hemoglobin, causes red blood cells to adopt a sickle-shape, restricting oxygen flow throughout the body.2 Over the past decade, outcomes have improved with the introduction of therapies like Endari (Emmaus Life Sciences) and Adakveo (Novartis). However, progress has not been without setbacks, and substantial unmet needs remain. Now, with gene therapies Casgevy (Vertex/CRISPR Therapeutics) and Lyfgenia (bluebird bio) available for over a year, access barriers remain a major hurdle, but encouragingly, referrals for these potentially curative options are steadily increasing. Spherix Global Insights recently released its latest findings from Patient Chart Dynamix: Sickle Cell Disease 2025 (US), an independent chart audit analyzing 187 SCD patient charts gathered from 109 US-based hematologists. This comprehensive review reveals continued unmet need despite hematologists' increased use of novel options, with half of patients having experienced two or more vaso-occlusive crises in the last year, and a majority reporting associated fatigue, pain, or anxiety. Given the significant burden SCD places on patients, hematologists are optimistic for a future with more disease-modifying therapies. One leading KOL affirms that for many of these patients, 'long-term, gene therapy is the solution'. Just over half of surveyed physicians surveyed have already referred a patient for gene therapy, or in some cases, administered it themselves. However, substantial logistical hurdles remain, with the majority of physicians believing that cost will continue to be a major barrier to widespread adoption. Despite these challenges, physicians are pressing ahead, confident that most patients are strong candidates for at least a discussion about gene therapy, whether now or in the future. Looking ahead, new therapies – including options beyond gene therapy – are in development, promising to further shift the treatment paradigm and improve outcomes for SCD patients. More than one-third of hematologists reported awareness of products in the pipeline, with particular enthusiasm for agents such as mitapivat (Agios), inclacumab (Pfizer), and BEAM-101 (Beam Therapeutics), among others. Physicians anticipate that once these therapies become commercially available, eligible patients will be initiated on treatment relatively quickly. This optimism is, however, tempered by the persistent challenges of cost and access that have complicated prescribing and referral patterns for currently available therapies. This comprehensive audit is the latest in Spherix's growing library of in-depth, data-driven insights in hematology. As the market continues to shift, and potentially curative therapies emerge, capturing the right prescriber and patient audience is paramount. Through rigorous independent research, Spherix provides this strategic insight to help pharmaceutical and biotech companies navigate the increasingly complex sickle cell disease market, reach the right patients, and ultimately improve both survival and quality of life. Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the 'why' behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists' attitudinal & demographic data to highlight differences between stated and actual treatment patterns. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix's unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit or connect through LinkedIn. For more details on Spherix's primary market research reports and interactive dashboard offerings, visit or register here: NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same.
Yahoo
17-04-2025
- Business
- Yahoo
Bluebird bio reaffirms Carlyle deal as Ayrmid misses deadline
Bluebird bio (bluebird) has reaffirmed its support for a proposed acquisition by Carlyle and SK Capital after rival bidder Ayrmid failed to deliver a binding offer or secure financing by the extended deadline. The announcement marks the second time Ayrmid has initiated discussions with bluebird without producing a fully financed proposal. Bluebird said it had engaged with Ayrmid for three weeks following the private equity firm's unsolicited bid in March, including extending the original deadline to allow more time for due diligence and deal structuring. However, Ayrmid did not submit a definitive proposal by the agreed-upon 15 April cut-off date and has yet to secure the necessary capital to proceed. As a result, bluebird's board of directors is reiterating its unanimous support for the acquisition agreement with Carlyle and SK Capital, which was first announced in February 2025. The board is urging shareholders to tender their shares by the updated deadline of 2 May, extending the previous expiration date of 18 April. bluebird's board chairman Mark Vachon said: 'Ayrmid's proposal remains highly conditional. bluebird has engaged with Ayrmid on two separate occasions—neither of which has resulted in a binding or fully-financed offer. The board unanimously reaffirms its support of the previously announced agreement with Carlyle and SK Capital in the strongest possible terms.' Bluebird has been under significant financial pressure for several years. The company currently faces the risk of defaulting on a $175m loan from Hercules Capital, secured in 2024 to support ongoing operations. Without a new capital injection, the board said, the company's liquidity position remains precarious. Carlyle and SK Capital's proposal offers $3 per share in cash, along with a potential additional payment of $6.84 per share if certain clinical and commercial milestones are met. Ayrmid's offer, disclosed last month, proposed $4.50 per share upfront and the same milestone-based payout structure. However, bluebird confirmed that Ayrmid has not demonstrated access to committed funding. bluebird noted that Ayrmid is continuing to seek financing and may provide further updates in the coming days. bluebird's financial position has deteriorated over time, despite achieving several key regulatory milestones. The company currently markets three US Food and Drug Administration (FDA)-approved gene therapies – Zynteglo (betibeglogene autotemcel) for transfusion-dependent beta-thalassemia, Skysona (elivaldogene autotemcel) for cerebral adrenoleukodystrophy, and Lyfgenia (lovotibeglogene autotemcel) for sickle cell disease – but has struggled with slow uptake, high costs, and reimbursement hurdles. In 2023, the company suffered a major setback when the FDA denied a priority review voucher (PRV) for Lyfgenia. Bluebird had already arranged to sell the voucher to Novartis for $103m, but the agency ruled that the therapy's active ingredient had previously been used in Zynteglo, for which the company had already received a PRV in 2022. The loss of the expected funds deepened bluebird's financial strain. To fill the gap, bluebird pursued emergency financing through a $150m public stock offering underwritten by Goldman Sachs and JP Morgan, as well as up to $100m in receivables financing from Alterna Capital Solutions. The company has also undergone repeated cost-cutting efforts, including workforce reductions of 30% in 2022 and an additional 25% last year. Despite these efforts, bluebird's therapies continue to face stiff competition. Lyfgenia is priced at $3.1m per patient, while a competing treatment, Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics, is listed at $2.2m. Both therapies were approved on the same day in December 2023 to treat sickle cell disease. Going public in 2013, bluebird has seen its stock decline sharply in recent years. Following the 16 April update, shares closed down by 7%. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. "Bluebird bio reaffirms Carlyle deal as Ayrmid misses deadline" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Reuters
29-03-2025
- Business
- Reuters
Bluebird bio receives non-binding bid for up to $110.5 million
March 28 (Reuters) - Gene therapy maker bluebird bio (BLUE.O), opens new tab said on Friday it had received a non-binding offer from peer Ayrmid for up to $110.5 million, sending its shares up 9.8% at $4.40 in extended trading. Bluebird bio, which has been facing a cash crunch, first raised going-concern doubts three years ago. It laid off a quarter of its workforce last year to focus on launching three gene therapies, including Lyfgenia for sickle cell disease, which has had slow uptake. Keep up with the latest medical breakthroughs and healthcare trends with the Reuters Health Rounds newsletter. Sign up here. Ayrmid, the parent company of cell therapy developer Gamida Cell, offered to acquire bluebird for $4.50 per share upfront, totalling about $43.8 million, according to Reuters calculations using data compiled by LSEG. The privately held UK company also offered an additional $6.84 per share, representing about $66.6 million, contingent upon bluebird achieving certain sales milestones. In February, bluebird bio said it had agreed to be taken private by Carlyle Group (CG.O), opens new tab and SK Capital Partners, for $3 per share upfront along with the same $6.84 per share, contingent upon it achieving certain sales milestones. Bluebird said its board is reviewing the Ayrmid proposal in consultation with its legal and financial advisors, and the board has not changed its recommendation in support of the Carlyle and SK Capital Partners deal.
CBS News
13-03-2025
- Health
- CBS News
Long Island man is first in New York history to be cured of sickle cell anemia
A patient on Long Island is the first in New York to be cured of sickle cell anemia, doctors say. They credit state-of-the-art genetic treatment. Medical history made at Cohen Children's Medical Center For 21 years, Sebastien Beauzile suffered chronic pain from debilitating sickle cell anemia. "Sickle cell was like a blockade for me, but now it's just like a wall that I just jumped over," he said. He is now making medical history in New York state. "The cliche 'the future is here' is actually true in this case," said Dr. Charles Schleien, with Cohen Children's Medical Center. "We feel blessed to be able to offer, be the first ones to be able to offer this," Dr. Jonathan Fish added. Groundbreaking Lyfgenia treatment used Beauzile's own bone marrow in IV transfusions to create normal red blood cells. "When we could use Sebastien's own stem cells to do this therapy, we were delighted," Dr. Banu Aygun said. "Sickle cell disease was described in modern medicine in 1910, and here we are over 100 years later, and this is the first cure you are seeing," Dr. Jeffrey Lipton said. "I feel unstoppable" In the United States, sickle cell amenia most commonly affects people of African, Mediterranean and Middle Eastern descent. Cohen Children's Medical Center hopes to target those communities with this life-altering therapy. "To the Med 4 team, the hematology team, the transplant team, you guys have really changed my life," Beauzile said. "Sebastien been coming to this hospital since he was 2 months old. There is not enough words to show you how grateful I am," said Magda Lamour, Beauzile's mother. Beauzile will soon be able to travel, work out and focus on his education. He hopes to work in the medical world. "Amazing, and I can't wait to get back to my day-to-day life because now I feel unstoppable," Beauzile said.
