Biogen Idec quietly carves out a leading role in developing gene therapy for hemophilia

Olivier Danos

Biogen Idec ($BIIB) has taken its next big step toward building its own gene therapy program for hemophilia A and B. The Big Biotech and its newly named in-house expert Olivier Danos have executed a research deal with the San Raffaele-Telethon Institute for Gene Therapy (TIGET) to develop what they believe promises to be a durable treatment for hemophilia.

It's not a big money deal and a preclinical pact like this won't get much attention outside the field, but Biogen Idec has been carefully knitting together programs that are likely to be in the next big wave of gene therapy work. New medicines for the disease have been coming onto the market that offer longer lasting treatment, but an effective gene therapy for hemophilia--inserting a corrective gene--would mark a dramatic leap forward for patients with the potentially lethal bleeding disorder. And the early-stage deals going on now could start to move very quickly in the clinic as big players invest heavily in the technology.

Biogen Idec--already a big player in hemophilia and a widely respected leader in MS drug development--is putting up a modest $5 million and two years of research support to get started. In turn it gets to work with TIGET's lentiviral vectors that specifically target liver cells.

Doug Williams

"That's the next generation, gene therapy," Biogen Idec R&D chief Doug Williams told FierceBiotech at the JP Morgan conference several weeks ago. In a preview of today's deal, Williams emphasized that the company wanted to get the right delivery vector, noting the large payload that they're working with.

Gene therapy has been booming over the past two years as developers like bluebird bio, partnered with Celgene ($CELG), have powered their way back in after the first generation of work was shelved following the emergence of lethal side effects. And Biogen Idec has some catching up to do as hemophilia has become a leading target for this new wave of biotechs. Spark Therapeutics, which is going public next week, is focused on hemophilia with Pfizer ($PFE). Other players include Dimension Therapeutics, ReGenX BioSciences and others.

They're all shooting for a durable cure. "People with hemophilia often require life-long treatment to control dangerous bleeding, but there is hope that gene therapy could one day lead to a single-dose, lasting therapy," said Danos in a statement. 

Danos was there at the beginning. He and James Wilson, another key player behind this new wave of gene therapy efforts, both worked at Richard Mulligan's lab in Harvard back in the '80s. He's since gone on to work at University College London and several biotechs before joining Biogen Idec. Now he'll be working closely with colleagues in Italy. And they'll be trying to distinguish their work with a benign lentiviral against the AAV tech in play at other companies.

"We at TIGET have worked for several years to develop a new vector design that upon administration into the blood stringently targets expression of its genetic cargo to the hepatocytes, the main cell type of the liver. This was crucial to establish long-term expression of the therapeutic gene and obtain proof-of-principle of its therapeutic benefit in experimental models of hemophilia B. We are now delighted to collaborate with Biogen Idec to expand our hemophilia gene therapy program also to hemophilia A and to advance both programs towards clinical testing, always keeping in mind the safety and benefit of the patients," said Luigi Naldini, director of TIGET.

- here's the release

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