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Next Stop on Father’s Duchenne Quest: Wall Street? Solid Raises $50M

Xconomy Boston — 

It wasn’t too long ago that Ilan Ganot was a J.P. Morgan banker. Then his son, Eytani, was diagnosed with Duchenne muscular dystrophy, and he ended up the head of a startup trying to find an array of treatments for the progressive, deadly genetic disease.

Now it appears that journey might return Ganot to a place he’s well familiar with, Wall Street—this time as the CEO of a publicly traded company.

Ganot’s Cambridge, MA-based company, Solid Biosciences, has raised a $50 million Series C round from a wide range of “crossover” investors who invest in both public and private companies, and existing backers including Boston area biotech giant Biogen (NASDAQ: BIIB).

The new round, led by RA Capital Management and Bain Capital Life Sciences, suggests that Solid has grown to the point that it is now at least considering an initial public offering. “We are in a strong capital position, but are keeping all of our options open,” Ganot says of Solid, which has now raised $115 million in since its inception in 2013.

Ganot formed Solid after his then two-and-a-half-year-old son, Eytani, was diagnosed with Duchenne. The progressive muscle disease affects roughly 300,000 boys worldwide and robs them of their ability to walk by their teens. Patients often die of lung or heart problems at an early age. There are now two approved drugs for Duchenne, eteplirsen (Exondys 51) from Sarepta Therapeutics (NASDAQ: SRPT) and a steroid, deflazacort (Emflaza) owned by PTC Therapeutics (NASDAQ: PTCT). But neither of these drugs are cures. Eteplirsen is supposed to slow progression but is only approved for a small fraction of people with the disease, while deflazacort helps boost muscle strength.

Ganot had been a London-based banker at J.P. Morgan, but after the diagnosis, he met with key players in the Duchenne field, researched, and ultimately left the banking business. His former employer led a Series A round for Solid to help get it off the ground.

As Ganot described to Xconomy in 2015, Solid was originally structured as an operating company with subsidiaries. “Unlike most biotech companies that are driven by a technology, anything goes with us,” he said at the time. “There’s nothing we don’t care about if it has a chance to affect Duchenne.”

So Solid formed three divisions: Solid One, which in-licenses drugs used for other diseases and tries them out in Duchenne; Solid GT, which housed a gene therapy treatment; and Solid Suit, for which the plan was to develop a “soft exo suit” that Duchenne patients could wear to help offset their use of muscles.

Solid still has all of those elements in place. It has a gene therapy program moving forward, three experimental drugs in preclinical testing, and a wearable device being developed with the help of Menlo Park, CA, nonprofit research organization SRI International. But in connection with the funding today, the company has unwound that complex corporate structure and merged all of its units together. “There were historical considerations that no longer apply,” Ganot says of the change.

Gene therapy—a method of using a virus to shepherd genetic instructions into the body—has been tried for Duchenne before, though never successfully. But Solid’s program is one of a new group of emerging gene therapy and gene editing programs being advanced by academic groups, startups, and larger companies. Sarepta, for instance, has cut deals for two gene therapy programs from Nationwide Children’s Hospital. Startup Exonics Therapeutics launched just last month to advance a gene editing treatment that originated at the University of Texas Southwestern Medical Center. Scientific groups at Duke University, Harvard University, UCLA and the Broad Institute of MIT and Harvard and others have been focused on gene editing as well. These programs all offer at least the potential for a one-time, long lasting treatment for the disease.

Solid’s gene therapy program, meanwhile, was licensed from the work of University of Washington neurologist Jeff Chamberlain and University of Missouri immunologist Dongsheng Duan. Duan’s team published a study in Human Molecular Genetics in early 2015 showing the delivery of a Duchenne gene therapy in dogs.

That program has progressed the fastest for Solid, and was crucial in helping the startup bring Biogen and Perceptive Advisors aboard to lead a $42.5 million Series B round for the company in 2015. The gene therapy program, known as SGT-001, should begin clinical trials in the second half of this year. “We believe [it] has the potential to slow or even stop the disease in a majority of patients,” Ganot says.

Solid also plans to use the cash to develop more gene therapies and other experimental Duchenne drugs for its pipeline, both internally and through deals. “We are conducting plenty of R&D, and are also open to anything and everything that’s worth doing in the fight against [Duchenne],” Ganot says. “I’m proud of our growing team and believe in our ability to make a big difference for patients.”

RTW Investments, Foresite Capital, Cormorant Asset Management, Leerink Partners, and Janus Capital Management participated along with RA, Bain, and Biogen in Solid’s Series C round. RA Capital managing director Rajeev Shah and Bain Capital managing director Adam Koppel (formerly of Biogen) have joined Solid’s board.