The cash will allow the company to hire scientists to conduct preclinical research into a potential treatment for DMD.
Cambridge, Mass. – Exonics Therapeutics closed on a $40 million Series A yesterday. The financing was via The Column Group (TCG). In addition to the raise, TCG’s David Goeddel, managing partner, and JJ Kang, principal, will join Exonics’ board of directors.
The company plans to lease space in Cambridge and start hiring scientists to begin preclinical research. The company hopes to have the new lab operational in the early part of 2018. An as-yet unidentified scientist will lead the team at the facility, and the company’s recruiters have begun looking for talent.
Exonics focuses on developing SingleCut CRISPR technology to fix mutations that cause Duchenne muscular dystrophy (DMD) and other neuromuscular diseases. DMD is a muscle-wasting disease that primarily affects young boys, leading to muscular weakness and eventual death, often in their twenties. It is caused by mutations in the DMD gene that prevents the production of dystrophin, a key protein that helps stabilize and protect muscle fibers.
John Ripple, the company’s chief executive officer, told Endpoints News, “With Dr. Olson and his team’s expertise there at UT Southwestern, and with the team that we’ve been able to build here at Exonics, and now the funding that we have, we think we’re very well positioned to at least be a leader in this field, and to hopefully be the first, or one of the first, to bring promising treatments that will address the cause of Duchenne muscular dystrophy for these boys.”
In a statement, Goeddel said, “The company has generated compelling early data, and we are pleased to support Exonics as it advances its preclinical development program in Duchenne closer to the clinic.”
Currently, Sarepta Therapeutics and its Exondys 51, are the leader in DMD treatment. Exondys 51 had a dramatic and still controversial journey to approval, and the drug’s efficacy is still in question. It also only is approved for use in a subset of the DMD population that makes up about 13 percent of patients.
Exonics has published preclinical data in mice that suggest its approach might be able to repair the mutations in the gene. Exondys 51 uses so-called “exon skipping” technology, which is a partial gene therapy that introduces a truncated piece of the gene. DMD is caused by mutations in the DMD gene, which involves muscle development. It is the second-largest gene in the human genome, and because of its size, it’s ineligible for current approaches using adeno-associated virus (AAV) as a vector. As a result, Exondys 51 only appears to increase dystrophin production by about 1 percent.
Amber Tong, writing for Endpoints News, notes, “Since Exonics launched in February, and even before, other companies have been developing their own gene therapy for DMD. Sarepta—which sells controversial Exondys 51—announced a couple weeks back that it’s partnering with Duke to explore its own CRISPR technology. This highlights the urgent unmet need for improved DMD treatment, and while Ripple acknowledges that, he said Exonics wants to ensure they have it right.”
“We are trying to develop an optimal treatment for these boys to make sure that we have the best possible treatment when we do enter the clinic and give that first administration to these boys,” Ripple told Tong.
