Ilan Ganot never planned a career in drug development. But everything changed for him and his wife, Annie, when their son, Eytani, was diagnosed two years ago with a rare disease.
"Even just looking at Wikipedia, you see wheelchair by 10, 11, 12, and that most of the boys don't make it past their early 20s," Annie said in an interview. "It was devastating. Words can't describe, as a parent, how you feel."
Eytani, 4, has Duchenne muscular dystrophy, a genetic muscle-wasting disease. There's no cure for DMD, and no drugs are approved for it in the U.S.
Ilan, who was working with hedge funds at JPMorgan when Eytani was diagnosed in October 2012, decided to change that. After quitting his job, moving the family to the Boston area—a hub for biotechnology research—and raising $17 million, he founded Solid Ventures. It's a for-profit company focused solely on Duchenne. It aims to step in where traditional drug companies sometimes won't go.
"In rare diseases—and there are thousands of them—we find it difficult sometimes to substantiate the economic value, to justify for big companies to roll up their sleeves and jump right in," Ganot said. "What we're left with is a lot of patients that are left without drugs, and a lot of science that is not getting developed."
Solid—so named for the English translation of Eytani's name from Hebrew—aims to take promising science out of labs or off pharmaceutical companies' shelves and advance it through development. The company has now made its first moves, delving into three initiatives to treat DMD in different ways.
The first draws a pharmaceutical giant into the fray. Solid is collaborating with Pfizer to test one of its clinical assets, with plans to decide whether to move into human testing by early next year. Ganot declined to name the therapeutic area in which Pfizer had been focusing the drug, but said it may be possible to move directly to the mid-stages of clinical testing because enough is understood about the molecule.
Solid's second approach could be more transformative for DMD—if it's successful. The company has formed a subsidiary focused on gene therapy called Solid GT, which "if it works changes the disease completely," Ganot said. "It's relevant for every patient."
Gene therapy involves inserting a gene into patients' cells, usually using a virus as a delivery method. Progress in the technology has been halting but has started to accelerate in recent years, with companies including Bluebird Bio and Sangamo showing some early success.
Solid GT is guided by a scientific advisory board chaired by the University of Pennsylvania's James Wilson, Ganot said. Its goal is to take a smaller form of the gene dystrophin—the key to DMD, and also the largest known human gene—and administer it to patients so it can help them make a smaller version of the protein important for muscle function, also called dystrophin.
Ganot said the company expects to be in human trials with the gene therapy approach within two years.
The third initiative, called Solid Suit, focuses on preserving muscle function, "repurposing military technologies for the benefit of health care and patients with disabilities," Ganot said.
"DMD is a muscle-wasting disease, and when the muscle works, it gets wasted, basically, and doesn't regenerate like it does in healthy people," Ganot explained. "We believe that if we could preserve much of that muscle, when disease-modifying interventions like exon-skipping or gene therapy are introduced, the more muscle we have left, the better chances of an efficacious treatment."
Exon-skipping is a technology being pursued by Sarepta Therapeutics and Prosensa, both aiming to file for approval of their drugs by the end of this year. Their medicines focus on treating specific mutations that drive DMD, and they're both starting with the one that affects the most patients—not the one that Eytani has.
Solid's multiple approaches to one disease are part of its strategy to diversify its efforts to lower investment risk, Ganot said. They're also structured in ways that provide the most flexibility to the programs. Solid GT is a subsidiary, which "gives us better options in the future on partnering, raising specific money for a particular program, on discontinuing or funding some more," Ganot said.
"All three would help all patients," he said. As for his own son, Ganot said he's confident Solid will make a difference.
"We're aggressive, we raised enough money to do what we need to do, we have the right people, and very soon we'll have the right treatments, too."
—By CNBC's Meg Tirrell