Chicago biotech argues bluebird, Third Rock 'killed' its rival, pioneering thalassemia gene therapy in lawsuit
Bluebird bio $BLUE chief Nick Leschly courted controversy last week when he revealed the company’s beta thalassemia treatment will carry a jaw-dropping $1.8 million price tag over a 5-year period in Europe — making it the planet’s second most expensive therapy behind Novartis’ $NVS freshly approved spinal muscular atrophy therapy, Zolgensma, at $2.1 million. A Chicago biotech, meanwhile, has been fuming at the sidelines. In a lawsuit filed earlier this month, Errant Gene Therapeutics alleged that bluebird and venture capital group Third Rock unlawfully prised a viral vector, developed in partnership with the Memorial Sloan Kettering Cancer Center (MSK), from its grasp, and thwarted the development of its seminal gene therapy.
Errant Gene Therapeutics was formally established in October 2003 by Patrick Girondi, whose son Rocco was two years old when he was diagnosed with a severe form of the rare, inherited blood disorder. In 2000, Errant contacted an MSK scientist, Michel Sadelain, who had published a paper exhibiting the potential of his gene therapy technology in mice — and began to fund Sadelain’s research. Five years later, Errant purchased from MSK the exclusive, global rights to harness Sadelain’s technology to work on gene therapies for blood disorders: beta-thalassemia and sickle cell disease. In the years that followed, Errant worked with MSK to create a vector — which was then patented by the company in the United States in 2009, the lawsuit said.
In late 2016, Errant alleged it discovered that bluebird and Third Rock — a firm which Leschly was a founding member and a former partner of — were in cahoots to obstruct its gene therapy from reaching the market by engaging in a deliberate pattern of fraud and deception, and indicative of “a troubling pattern of tortious business practices undertaken by pharmaceutical companies to secretly and systematically ‘kill’ competing medical treatments.”
Back in 2010, Errant was ready to initiate clinical trials with MSK and the NIH. At the time, the other drug developer with an experimental thalassemia gene therapy was Genetix Pharmaceuticals. That year, Third Rock invested in Genetix, installed Leschly as chief, and switched the company’s name to bluebird bio.
According to the lawsuit, Third Rock then met with MSK and “admitted” that the Errant vector outshined bluebird’s vector — and was advised to approach Errant about buying its technology. When Third Rock did not agree to abandon the bluebird vector in favor of Errant’s, the plaintiff asserted that bluebird and Third Rock moved to snuff the development of the Errant vector by accessing the technology via a “secret agreement” with MSK. In addition, bluebird and Third Rock relied upon their relationships with newly crowned MSK chief Craig Thompson to further stall the development of Errant’s gene therapy, the lawsuit added.
Later in 2010, MSK demanded that Errant find a commercial partner as a condition before starting clinical trials — but despite Errant fulfilling that condition, MSK rejected the contract and instead initiated arbitration against Errant, the drug developer claimed in the lawsuit. Eventually by 2011, Third Rock and MSK “convinced” Errant to give MSK its vector in exchange for a 50% royalty for any product developed using it, Errant said, suggesting it is owed direct damages to the tune of “hundreds of millions of dollars.”
A Third Rock spokesperson said it had no comment, while a bluebird spokesperson said the company does not comment on litigation.
This is not the first time Errant’s Girondi has lashed out at MSK. A report by the New York Times in 2015 chronicles Girondi’s previous attempt. MSK was forced to take back control of the project because Girondi — a high school dropout who was arrested various times in his youth, but dodged prison by joining the Air Force — lacked the”necessary money and the expertise,” the report said, citing Andrew Maslow, the director of industrial affairs at MSK until early 2012.
Endpoints News has contacted MSK for comment.
Earlier this month, bluebird’s Zynteglo — a one-shot gene therapy engineered to replace a lifetime of transfusions and medication for patients aged 12 years and older — was approved in Europe. While the Cambridge, Massachusetts-based company has produced persuasive data — dramatically reducing the need for blood transfusions in a 22-patient study — the gene therapy has fallen short of a cure in severe patients. Phase III trials designed to confirm Zynteglo’s benefit are ongoing, and US approval is anticipated next year.