Biogen Helps Put $42.5M Into a Father’s Duchenne Gene Therapy Quest

Xconomy Boston — 

Duchenne Muscular Dystrophy is a progressive muscle wasting disease that affects about 300,000 people, primarily boys, around the world. Patients with the disease lose ability to walk by their teens, and often die from one of a number of complications—like respiratory or heart failure—at a young age. There is no cure and no effective treatment.

Ilan Ganot knows this story all too well. His son, Eytani, was born with the crippling disease. So the former J.P. Morgan banker started a company, Solid Biosciences, to fight it, and he just raised a bunch of money to move a potential gene therapy for the disorder into clinical testing.

The money, a $42.5 million Series B round from Biogen (NASDAQ: BIIB), New York’s Perceptive Advisors, funds managed by Janus Capital Management, and other unnamed private investors, isn’t actually going to Solid itself.

That’s because Cambridge, MA-based Solid is the parent of three subsidiaries—Solid GT, Solid One, and Solid Suit—each pursuing a different strategy to help impact Duchenne. Solid GT houses the gene therapy program being carried forward, a treatment that Ganot (pictured above, with Eytani) aims to get into clinical testing in 2017, and that’s the company that has gotten the Series B cash.

Gene therapy—a method of using a virus to shepherd genetic instructions into the body—has been tried for Duchenne before, but never successfully. Ganot believes key scientific advances will make a difference this time.

The first advance is the delivery vehicle, or “vector.” The University of Pennsylvania’s James Wilson, a gene therapy pioneer and co-founder of gene therapy companies RegenXBio and Dimension Therapeutics, chairs Solid GT’s scientific advisory board. Wilson played a big part in advancing the use of so-called adeno-associated virus (AAV) vectors, which have become a gene therapy workhorse. Solid is using AAV vectors to deliver the therapy, though Ganot wouldn’t specify if these vectors are licensed from Wilson’s work or RegenX.

The second piece is the gene the AAV vector will deliver. Ganot says this will be what’s known as “micro-dystrophin.” He calls it a “very well thought-through, rationally designed sequence” that is small enough to pack into an AAV, yet carries “all of the critical pieces of the dystrophin gene.” Duchenne patients lack dystrophin, a key protein that keeps muscles intact, and that’s why their muscles progressively get weaker. But a full-sized engineered dystrophin gene is too large to fit into an AAV, so it couldn’t be effectively used—hence trying to use a smaller one that can still provide the same benefit.

The idea—and a big challenge, for sure—will be to deliver this therapy systemically, and in large amounts, through an intravenous infusion. It will circulate through the body, with the goal of expressing the micro-dystrophin protein in muscle cells. One of Solid GT’s scientific advisory board members, University of Missouri professor Dongsheng Duan, recently completed a study published in Human Molecular Genetics showing the delivery of a Duchenne gene therapy in dogs. Duan’s technology was licensed to Solid GT, and that work helped the company get its investment from Biogen and Perceptive, according to Ganot.

Ganot’s story, first reported by Bloomberg in February of last year, is an unusual one. He was a banker for J.P. Morgan in London when Eytani, then two and a half years old, was diagnosed with Duchenne. Ganot was devastated.

“[Duchenne] is a disease that leaves very little room for hope, it’s a very long, steady and devastating decline,” he tells Xconomy. After the diagnosis, “nothing really changed, except everything changed—even if it hadn’t actually changed yet,” Ganot says.”Your kid is just as good as [he was] the day before the diagnosis, except you know what’s coming.”

Ganot took time off work, helped take his kids to school (he has three children), and did “what I think a lot of parents do when these things happen, which is to try to figure out what’s going on.” He researched, met with key players in the Duchenne field, and decided to start a company entirely focused on Duchenne treatments. Though he left J.P. Morgan to do it, his former employer led a $17 million Series A round for Solid.

“The plan was to go and get the best expertise you could have in just one problem and figure out how to solve it,” he says. “Unlike most biotech companies that are driven by a technology, anything goes with us. There’s nothing we don’t care about if it has a chance to affect Duchenne.”

That’s why Ganot has structured Solid as an operating company with subsidiaries. Solid One is in-licensing drugs used for other diseases and trying them out as potential treatments for certain symptoms of Duchenne. It has deals in place with Pfizer and Swiss biotech Debiopharm, for instance, to test out drugs for Duchenne. Ganot won’t divulge many details about the Pfizer drug, but the Debiopharm compound is known as alisporivir, and was once tested by Novartis in more than 1,500 patients as a potential treatment for hepatitis C.

With Solid Suit, meanwhile, Ganot aims to develop a “soft exo suit” that Duchenne patients could wear to help offset their use of muscles. Ganot notes that as Duchenne patients use their muscles over time, they break down. The idea would be for a suit to make, for instance, 1,000 steps feel like 600. “Maybe we can save the other 400 steps for tomorrow,” he says. “This is a battle against time after all.”

Solid GT, however, has attracted the most attention and funding. It started with a $10 million Series A last year that partly came from three non-profit institutions—the Duchenne Research Fund, Duchenne Children’s Trust, and Joining Jack, according to Ganot. Now Biogen and others have jumped in.

“My son is the reason I’m doing this, but I don’t do it just for him,” Ganot says. “At this point I think what we’ve put in place gives us a really good chance to make a major difference for every patient. And that’s really what we’re after.”

Perceptive’s Adam Stone and Biogen’s Lynne Sullivan have joined Solid GT’s board as part of the funding.