Exonics Therapeutics, one of the players in an emerging race to use gene therapy or gene editing techniques to treat Duchenne muscular dystrophy, has just landed a big round of funding to take its first treatment into clinical testing.
The Cambridge, MA, company, seeded last year by a subsidiary of the nonprofit group CureDuchenne, will use a $40 million Series A round led by The Column Group to advance a Duchenne treatment using the gene editing technology CRISPR-Cas9.
Patients with Duchenne, who are primarily boys, lose the ability to walk by their teens, and often die from complications like respiratory or heart failure at a young age. Over the past year, two treatments have been approved—eteplirsen (Exondys 51), from Sarepta Therapeutics (NASDAQ: SRPT), and deflazacort (Emflaza), now owned by PTC Therapeutics (NASDAQ: PTCT)—but neither are cures. Sarepta’s drug is approved for a subset of Duchenne patients, roughly 13 percent with a specific genetic malfunction, for which it is supposed to slow the progression of the disease. Deflazacort is a muscle-boosting steroid already widely available in other countries, also meant to help slow Duchenne.
Companies and academic institutions have been increasingly investing in gene therapy and gene editing techniques, however, hoping for a potentially much longer lasting solution. The methods would supply genetic instructions for a patient’s body to produce a muscle-protecting protein, dystrophin, that people with Duchenne lack.
Two Boston-area companies, for instance, are on the verge of advancing experimental gene therapies for Duchenne into clinical testing. By the end of 2017, Sarepta will begin Phase 1 studies for two different gene therapies that originated at Nationwide Children’s Hospital in Cleveland. Solid Biosciences, a privately held company that raised $50 million earlier this year,…