Vertex Acquires Exonics Therapeutics, Advancing Gene-editing Treatment for DMD

Biotechnology company Vertex has acquired Exonics Therapeutics, which focuses on using gene-editing technologies to treat Duchenne muscular dystrophy (DMD) — a move that is being hailed as a positive step toward getting these therapies into clinics.

Exonics was co-founded in 2017 by CureDuchenne, a nonprofit organization focused on improving care and building community for people with DMD, and Eric Olson, PhD, a leading researcher in the gene-editing technology called CRISPR/Cas-9.

“We are thrilled to work with Exonics and the entire Duchenne community to advance the promising science that Eric and his team have developed to change the course of this devastating disease,” Jeffrey Leiden, MD, PhD, president and CEO of Vertex, said in a press release.

Using a system modified from bacteria, CRISPR/Cas-9 allows researchers to remove or insert small pieces of DNA in genes, a process broadly called gene editing. Olson and his colleagues have published several studies demonstrating that this technology can functionally correct mutations in the dystrophin gene, which cause DMD, in both mice and human cells.

More specifically, the insertions and deletions made by the scientists cause the DNA-reading machinery of the cell to skip certain protein-encoding segments, called exons, of the dystrophin gene when it is “read.”

DMD-causing mutations tend to cluster in some of these exons. By skipping them, the cell is able to make a version of the dystrophin protein that, while still imperfect, functions sufficiently. The strategy has been compared to putting a spare tire on a car: not ideal, but good enough to get you where you’re going, the researchers said.

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To Olson, the acquisition of Exonics by Vertex is a sign that this preliminary data has a real chance of being translated into clinical use to improve the lives of people with DMD.

“CureDuchenne believed in the science and our team early on and enabled Exonics to show significant proof of principle for gene editing in Duchenne. We are thrilled to work with a large biotech company that can help us move our science from preclinical to clinical as fast as possible,” he said.

CureDuchenne is equally excited about the acquisition.

“We could not be more thrilled about the partnership between Exonics and Vertex. Vertex has an incredible track record in accelerating drug development for rare diseases and they are exactly what the Duchenne community needs to develop and test gene-editing treatments for Duchenne as quickly as possible,” said Debra Miller, founder and CEO of CureDuchenne.

“This announcement gives the entire Duchenne community a reason to celebrate because we are getting closer to a cure for Duchenne,” Miller added. “We are grateful to Dr. Eric Olson and the team at Exonics for their hard work in achieving impactful results in their preclinical research and we look forward to continued and expedited successes with Vertex.”