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: [[ticker: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
