UPDATED: Biogen enlists gene therapy stars, adds vectors in $2B deal

Biogen
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Ten months after Biogen signed off on a $1 billion-plus deal to partner with AGTC on a slate of gene therapy programs for rare eye diseases, the Big Biotech is taking another big step forward in the field, allying itself with two of the world’s preeminent experts in gene therapies while expanding its focus into gene editing and picking up some tailor-made vector tech in a pact with Regenxbio ($RGNX).

Biogen ($BIIB) struck a collaborative deal to expand its work in gene therapies and gene editing with James Wilson and Jean Bennett, two longtime experts in the field, which will be worth up to $2 billion for the University of Pennsylvania. The pact starts with a $20 million upfront, includes $62.5 million in research support with up to $137.5 million in milestones for each of 7 preclinical R&D programs and a set of options for next-gen vectors from the two investigators.

The pact covers new work on gene therapies for the eyes as well as skeletal muscle and the central nervous system. 

Bennett is responsible for groundbreaking research in delivering a gene therapy to cells in the retina. In recent years she has also helped spawn and advise on clinical programs for a variety of biotechs, including France’s GenSight and Spark Therapeutics.

“Gensight is focusing on optogenetics and Leber's hereditary optic neuropathy,” Bennett tells me in an email. “The Biogen support will target retinal degenerative diseases caused by single gene defects. The Regenx vectors are more efficient at targeting photoreceptors than AAV2, the vector that has been used most commonly so far in gene therapy clinical trials targeting blinding disease.”

The Regenxbio platform has spawned some 100 vectors altogether, each with a particular advantage in enhancing gene expression for particular targets. And while Regenxbio now has its own pipeline, it’s been financing operations with the help of a string of licensing deals like this, making the company into something close to a central warehouse for AAV vectors. Biogen gained rights to their AAV8 and AAV9 vectors, which Regenxbio says should help enhance the intended therapeutic impact in the eye.

Biogen has been steadily upping its investment in gene therapies, signaling its big move into the field with its decision to hire Olivier Danos in 2014 to head up the operation. It was a particularly astute choice, as what you know in gene therapy closely correlates with who you know. 

Danos has spent decades in the field, working with Wilson in Richard Mulligan’s Harvard lab back in the ‘80s. Now, Danos will be working closely with Wilson again while using delivery vectors from Regenexbio that Wilson helped to create. 

Regenxbio counts Wilson, a pioneer whose early trial ended in the death of a patient and a big chill for gene therapy that lasted for years, as a scientific co-founder. Over recent years, as the safety issues and AAV vectors became better understood, gene therapy research and development exploded. Regenxbio became something of a clearinghouse for AAV tech in the process, licensing out vectors to biotechs like AveXis and Voyager and co-founding Dimension.

“This is our 9th license,” Regenxbio CEO Ken Mills tells me. And while he’s not currently able to spell out the financial details in the deal, he adds that you can expect that they all conform to a roughly similar set of terms on upfronts and milestones.

Biogen picked up a clinical-stage program and several preclinical projects in its AGTC deal last year. But it now plans to get more ambitious.

“This is a new area for (Biogen) in terms of these indications,” says Mills. “This was Biogen wanting to work on its own programs, with the best people and best technology.”

Biogen has built a reputation for undertaking ambitious research efforts. But over the past year its once-sterling rep on Wall Street suffered as MS revenue from its blockbuster Tecfidera franchise began to falter. The Biogen team, led by CEO George Scangos, reacted instantly, carving out hundreds of jobs to help reassure Wall Street it will continue to deliver. For many analysts, Biogen’s R&D division is taking some home run swings at Alzheimer’s and more, but won’t be able to connect with any new markets for some time, at best. And that’s causing some anxiety about a shortage of near-term catalysts that an early-stage effort on rare eye diseases will do little to alleviate.

“Biogen views the development of gene editing technology as a natural extension of its current efforts to develop gene therapies employing viral and non-viral vectors,” noted Danos in a statement. “The successful development of gene editing technology for the treatment of inherited or acquired diseases requires expertise not only in a broad range of biological disciplines, such as translational development and viral and non-viral delivery, but also in a variety of aspects of conventional pharmaceutical product development, including manufacturing and clinical development.”

- here's the release

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