Biogen Turns To UPenn Gene Therapy Pioneers in Wide-Ranging Alliance

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$62.5 million in research funding over the next three to five years in the alliance. UPenn could receive $2 billion total if all seven programs hit a variety of milestones, an extremely unlikely scenario (which is why future dollar figures attached to longterm biotech deals are often known as “biobucks”).

Separately, Biogen has also cut a deal with RegenXBio to use two types of AAV viruses—AAV8 and AAV9—for some of these programs.

The deal has both near-term and long-term implications for Biogen. According to Olivier Danos, who leads Biogen’s two-year-old gene therapy division, the programs for which Biogen will use AAV8 and AAV9 could be ready for clinical testing within two years. These programs will be gene therapies for rare forms of inherited blindness, achromatopsia and choroideremia. Danos says Biogen will also fund UPenn’s work on a gene therapy for spinal muscular atrophy. He didn’t provide details about the other four programs in the collaboration.

Biogen is already working on two gene therapies for two other rare forms of inherited blindness with Gainesville, FL-based AGTC (NASDAQ: AGTC). The company also formed a collaboration last year with two Italian entities on a gene therapy for hemophilia, but announced plans earlier this month to funnel that work into a new hemophilia spin-out.

In the UPenn deal, meanwhile, Biogen will also lean on Wilson and Bennett for what it calls in its announcement “next-generation gene transfer technology.” The two researchers are developing newer types of AAV viruses meant to be more efficient at delivering genes into patients’ cells than the AAV versions currently available. Biogen isn’t funding this work, and won’t get exclusive licenses to these AAVs—which, it should be noted, are not owned by RegenXBio either—just an option to use them in programs Biogen takes forward. Wilson, Danos says, is also collaborating with other Penn researchers on other delivery tools that aren’t viruses—things like phospholipids, which are molecules consisting of two fatty acids. Biogen is keeping an eye on the work. “This is how we get access,” Danos says.

This is all part of Biogen’s effort to possess improved gene therapy tools, like more efficient ways to deliver genes, or perhaps ways to, as Danos says, “control the amount of gene expression.” The gene therapies in clinical trials today are typically geared toward diseases characterized by a single genetic problem, like hemophilia, where one mutation prevents the production of a blood-clotting protein. Danos says developing better tools might help Biogen eventually go after more complex diseases of the brain or eye, such as multiple sclerosis, Parkinson’s, or age-related macular degeneration. That’s important for expanding the reach of gene therapy into more prevalent and complex diseases.

Biogen has other benefits in mind, too. The firm wants to get more involved in gene editing—a method of snipping out and replacing a defective gene.

Biogen already has a 2014 partnership with Sangamo Biosciences (NASDAQ: SGMO), which owns the rights to one method of gene editing known as zinc fingers. Biogen hasn’t yet cut a deal with any of the companies harnessing CRISPR-Cas9, a newer gene editing technology that has taken the scientific world by storm over the past few years. “This is obviously something we’re looking into,” Danos says, calling the UPenn alliance a “stepping stone” for a potential partnership with a CRISPR company, because it gives Biogen a deeper toolkit of delivery technologies that it and its partners can work with.

Likewise, Danos says, maybe smaller gene therapy developers might be able to benefit from Biogen’s help. Perhaps Solid Biosciences—a gene therapy startup that Biogen acquired a stake in last year—eventually needs some help moving forward a gene therapy it’s developing for Duchenne muscular dystrophy, Danos says.

“At some point if they’re gong to be successful in the clinic, they’re going to need to move to the next [step],” he says. “Who can do that? Probably not a small biotech.”

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