From High Hopes to Hard Times: The Fall of a Gene Therapy Pioneer

Bluebird Bio, once a shining star in the biotech world, is now a shadow of its former self. The company, known for its groundbreaking gene therapies, has been sold to private equity firms Carlyle and SK Capital for a mere $30 million. This sale marks the end of a long and tumultuous journey for Bluebird, which once had a market cap of around $9 billion. The company's downfall began in 2018 when a patient who received its gene therapy for sickle-cell disease developed cancer. Although Bluebird claimed its treatment wasn't the cause, the incident raised serious questions about the safety of its DNA-altering therapies. This was just the beginning of Bluebird's troubles. Bluebird also faced significant pushback from European payers after pricing its gene therapy for beta thalassemia, called Zynteglo, at $1. 8 million per patient. The company withdrew the treatment from Europe in 2021, just two years after it was approved there. Bluebird then shifted its focus to the U. S. , where it was preparing for the approval of Zynteglo for beta thalassemia, Lyfgenia for sickle cell disease, and Skysona for a rare brain disease called cerebral adrenoleukodystrophy.

Despite the approval of these therapies, Bluebird's financial struggles persisted. The company had been spending hundreds of millions of dollars a year, and offloading its cancer treatments into a new company, 2Seventy Bio, eliminated an important source of revenue. At last update in November, Bluebird said its cash would only fund the company's operations into the first quarter of this year. The sale to Carlyle and SK Capital is a stark contrast to Bluebird's past performance. The upfront price of about $30 million is a fraction of the $80 million Bluebird's former Chief Executive Officer Nick Leschly made from selling the company's stock during his time there. This sale raises questions about the viability of gene therapy companies and their ability to turn the promise of one-time treatments for rare diseases into profitable businesses. The entire field is facing tough questions right now. Vertex's competing gene therapy for sickle cell disease, Casgevy, has seen a similarly slow launch. Pfizer on Thursday announced it would stop selling a gene therapy for hemophilia that was approved only one year ago, citing weak demand. Bluebird's treatments could still change many lives. They just weren't enough to change the company's fate. The sale to Carlyle and SK Capital is a reminder that even the most promising biotech companies can face significant challenges. It's a cautionary tale for investors and patients alike, highlighting the risks and uncertainties in the gene therapy field.

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