Even though it recently decided to unload a gene therapy for hemophilia into a yet-to-be-named spin-out company, Biogen, one of biotech’s most risk-taking firms, is doubling down on gene therapy for other diseases. The Cambridge, MA-based company said today it is forming an alliance that could be worth $2 billion with two of the field’s pioneers at the University of Pennsylvania.
Jean Bennett and James Wilson have worked for decades of at UPenn on gene therapy, a method of ferrying genetic instructions into the body to produce what could become a long-lasting treatment for various diseases. Those effects are as yet hypothetical; no gene therapy has been approved in the U.S.
Bennett was one of the first to deliver a gene therapy into the eye, a program which Spark Therapeutics (NASDAQ: [[ticker:ONCE]]) of Philadelphia, which Bennett helped found, could carry forward to be the first ever gene therapy approval in the U.S. next year.
Wilson (pictured above), meanwhile, the director of UPenn’s gene therapy work, is a central figure in gene therapy’s three-decade-long rollercoaster ride. He co-led the infamous clinical trial that led to the death of teenager Jesse Gelsinger in 1999. He then became a key figure in advancing the gene therapy delivery tool known as an adeno-associated virus, or AAV, which has become a critical part of gene therapy’s renaissance. Wilson’s AAV work led to a company called RegenXBio (NASDAQ: [[ticker:RGNX]]), which has deals in place with a number of gene therapy players, among them Dimension Therapeutics (NASDAQ: [[ticker:DMTX]]) and Audentes Therapeutics.
Only now is gene therapy starting to inch towards the market. There are two approved gene therapies, both in Europe: Glybera, from Amsterdam’s UniQure (NASDAQ: [[ticker:QURE]]), for a rare metabolic disease; Strimvelis, from the British drugmaker GlaxoSmithKline (NYSE: [[ticker:GSK]]), for a rare immune system disorder.
Many questions remain about these treatments, like how long they will last and how much they should cost. Glybera, for instance, was launched with a $1 million price tag, and thus far, for a variety of reasons, as MIT Technology Review reported recently, has only been used on one patient. As Dimension CEO Annalisa Jenkins told Xconomy regarding Glybera earlier this year, the celebration over Glybera’s approval was short lived.
“The success of a therapeutic in a healthcare system is not the day it gets approved,” she said. “It’s the number of patients that it manages to impact and the value that it creates for shareholders.”
In other words, gene therapy is by no means a success yet. It won’t be until many patients on approved gene therapies are free of their disease for years, without complications. Potential treatments continue to advance, and new companies, such as the recent British startup Orchard Therapeutics, continue to launch. GSK won approval of Strimvelis in March. More data continues to accrue from Spark, UniQure, Bluebird Bio (NASDAQ: [[ticker:BLUE]]), BioMarin Pharmaceutical (NASDAQ: [[ticker:BMRN]]) and others in diseases like hemophilia, in which a person’s blood doesn’t clot properly and every cut or scratch could be life-threatening without treatment; cerebral adrenoleukodystrophy, when an abnormal immune response destroys myelin, the coating of the brain’s neurons; and Sanfilippo syndrome, an inherited metabolic disorder in which the body can’t break down certain types of sugar molecules. As Wilson told Xconomy in 2014: “I tell my wife that my career is starting at 60. That the field of gene therapy is now born. That it’s the beginning, it’s not the end.”
UPenn’s deal with Biogen (NASDAQ: [[ticker:BIIB]]) today is the latest example of significant investment in the field. Biogen will fund Wilson and Bennett’s work on seven different preclinical programs. Biogen will pay UPenn $20 million up front, and has committed another
