Biogen shoulders into gene therapy amid a second honeymoon for the field

Olivier Danos

As researchers and investors toast renewed optimism for a new generation of gene therapies, biotech bellwether Biogen Idec ($BIIB) is deepening its commitment to the space, another sign of life for the resurgent field.

The Big Biotech has hired Olivier Danos, most recently of Kadmon Pharmaceuticals, to serve as senior vice president in charge of its growing gene therapy operation, a position that reports to R&D chief Douglas Williams. Danos' job is to oversee Biogen's efforts to identify and develop new methods for gene transfer and genomic engineering, ferreting out potential deals across all of the company's therapeutic areas.

The overarching goal is to establish "a world-class gene therapy platform that combines our growing internal capabilities with collaborations with leading research organizations across the globe," Williams said in a statement, and the company hopes to grow a business unit that can stand alongside its existing efforts in antisense therapies, biologics and traditional small molecules.

For Biogen, the appointment spells a major vote of confidence in the growing maturity of gene therapy platforms, on the heels of a $320 million January partnership with Sangamo BioSciences ($SGMO), targeting two rare blood disorders through both gene replacement and editing.

And the company's interest is a big deal for gene therapy as a whole, which, thanks to the work of dedicated academics and a slew startups, has shaken off many of the deliverability pratfalls that doomed early research. After years of work, a new class of biotechs believes it has hammered out the right viral vectors to safely and predictably get corrective genes to their target tissues, spurring renewed hope for widespread clinical success.

However, Biogen is hardly the first major player to take notice. Last year, Celgene ($CELG) teamed up with bluebird bio ($BLUE) on a gene therapy approach to oncology, and, in 2014, Bayer signed a $252 million deal with Fierce 15 honoree Dimension Therapeutics to get its hands on a hemophilia A treatment. GlaxoSmithKline ($GSK), Baxter ($BAX) and others are working on in-house treatments in the field, as well.

But while Biogen may have some catching up to do, its history of moving swiftly on its key interests suggests a serious commitment to come. And though the company isn't disclosing any near-term disease targets in its new focus on gene therapy, Biogen's longtime devotion to hemophilia would make that indication a natural starting point. As programs at Dimension and elsewhere have demonstrated, monogenic forms of the rare bleeding disease have the potential to be cured by one-time treatments, and Biogen, which has mounted a multibillion-dollar effort to develop new therapies for hemophilia, has the expertise to scale up quickly.

And analysts say the move may be a prelude to a deal. As Piper Jaffray's Joshua Schimmer and Jerry Yang pointed out in a note to investors, as many as 7 gene therapies for hemophilia could enter the clinic by next year, and Biogen's competitors in the space have already snatched up rights to some of the most promising candidates. Getting serious about gene therapy could be Biogen's first step to taking action on an external program, the two write.

Whatever the result of Biogen's honed focus, Danos, who previously ran the gene therapy arm of University College London, believes the biotech has the right capital, intellectual and monetary, to stage some major advances in the expanding field.

"I have dedicated my career to advancing the use of gene therapy as a technology to develop lifesaving therapies for patients," Danos said in a statement. "The team at Biogen Idec shares this passion. It is their commitment to innovative science and willingness to tackle difficult medical challenges that attracted me to the company.

- read the statement

Suggested Articles

Fifteen of the 22 patients in a gene therapy trial no longer needed transfusions, while the remainder needed fewer transfusions.

Argos Therapeutics is ending its kidney cancer trial and mulling options, including a merger or sale, to stay alive.

CNS Pharma says berubicin is the first anthracycline drug to cross the blood-brain barrier and could transform treatment of the highly invasive brain tumor.