Vertex Pharmaceuticals is gearing up to tackle two types of muscular dystrophy with CRISPR gene-editing therapies. The Boston-based drug company will acquire the gene-editing start-up Exonics Therapeutics for $245 million and will pay Crispr Therapeutics $175 million to expand an existing collaboration.
In addition to the up-front payments, Exonics and Crispr Therapeutics each could earn $1 billion in milestone payments. Vertex will also launch a genetic therapies research site to conduct preclinical studies and manufacture CRISPR therapies for its clinical programs.
The news builds on announcements Vertex made in January to collaborate with the gene-editing start-up Arbor Biotechnologies on gene-editing systems that could be used for therapies. That month, Vertex also licensed two compounds from Merck KGaA that inhibit the DNA damage response, which Vertex believes may help boost the effectiveness of gene editing.
For a company that’s best known for its small-molecule drugs for cystic fibrosis, the investments signal Vertex’s ambition to claim its stake in the gene-editing field.
Vertex first dipped its toes into gene editing research in 2015 when it began collaborating with Crispr Therapeutics to develop permanent treatments for genetic disorders including cystic fibrosis and inherited blood diseases. The cystic fibrosis program is still in early preclinical research, but Vertex and Crispr Therapeutics are now testing a CRISPR gene-editing procedure to increase hemoglobin production in people with β-thalassemia. The duo plans on testing the same procedure in people with sickle cell disease too.
Treating those inherited blood diseases requires removing blood cells from patients, making a corrective edit with CRISPR, and reinjecting the altered cells for what will potentially be a long-term, or even permanent, treatment. Using CRISPR to treat muscular diseases will be more difficult, since the CRISPR will have to be delivered to muscle cells throughout the body.
Vertex now has obtained an exclusive license with Crispr Therapeutics to develop treatments for the most common form of muscular dystrophy, Duchenne muscular dystrophy.
Exonics, which launched in 2017 with $40 million, is also focusing on Duchenne. The start-up was founded by Eric Olson, whose lab at the University of Texas Southwestern Medical Center has used gene editing to demonstrate permanent treatments for Duchenne in isolated human cells, mice, and even dogs (Science 2018, DOI: 10.1126/science.aau1549). Exonics will become a subsidiary of Vertex and retain Olson as its chief scientific adviser.
Vertex and Crispr Therapeutics are also developing therapies for a second form of muscular dystrophy, called myotonic dystrophy type 1. And they are not alone in their ambitions to treat genetic muscle diseases. Sarepta Therapeutics has licensed CRISPR technology from Duke University researchers who used gene editing to treat muscular dystrophy in animals. Sarepta also acquired a small company called Myonexus Therapeutics that is developing gene therapies for several types of muscular dystrophy. And multiple companies, including Dyne Therapeutics and Avidity Biosciences, are developing oligonucleotide therapies for muscular dystrophies.