There’s still got a long way to go before we know if the landmark gene editing system known as CRISPR-Cas9 can really change how diseases are treated. But big biotech companies are now starting to dabble in it. The latest example: Vertex Pharmaceuticals has cut a deal with one of the field’s startups to try to develop drugs for crippling genetic diseases.
Boston-based Vertex (NASDAQ: VRTX) is paying CRISPR Therapeutics, a gene editing startup with offices in Cambridge, MA, and the U.K., $105 million—$75 million in cash, and $30 million via an equity investment—as part of a four-year research deal. The two companies will test out CRISPR-Cas9 therapies on a variety of diseases where the underlying targets have “been validated through human genetics,” according to a press release. Initially, that means cystic fibrosis, where Vertex already has an industry leading franchise, and sickle cell disease. Vertex has the option to license up to six treatments to come out of the deal. CRISPR Therapeutics could get up to $420 million in downstream payments for each of these programs, and royalties on sales, if these therapies ultimately move forward.
Vertex and CRISPR Therapeutics would split the costs and profits for any gene editing products targeting blood diseases. Vertex gets full rights for any other programs, such as a CF therapy, and will pay for the costs of discovery and development.
Vertex’s chief scientific officer David Altshuler (pictured above)—the famed geneticist and former deputy director and chief academic director at the Broad Institute of Harvard and MIT—will take a board observer seat at CRISPR Therapeutics as part of the deal.
In cases like CF and sickle cell, the two companies will try to use CRISPR-Cas9 to address the genetic mutations known to cause each disease. CF, for instance, is caused by mutations to the cystic fibrosis transmembrane conductance regulator, or CFTR gene. Vertex and CRISPR Therapeutics will try to correct those mutations with gene editing, or edit other genes that could contribute to the disease.
Sickle cell disease, meanwhile, has already become one of the first CRISPR-Cas9 disease targets. As Alex Lash reported earlier this year, scientists at the nonprofit Innovative Genomics Initiative—a University of California-funded group co-founded by CRISPR pioneer Jennifer Doudna—have been trying to cure sickle cell in mice with CRISPR technology. Vertex and CRISPR Therapeutics will look into gene-based treatments for other so-called hemoglobinopathies, conditions that lead to abnormal hemoglobin molecules, as well.
The deal is the latest CRISPR alliance to come together over the past few years as the technology—a method of genetic surgery derived from a bacterial defense system—has advanced. Novartis has aligned with another CRISPR startup, Intellia Therapeutics. Juno Therapeutics is working with Editas Medicine on T cell therapies.
These deals have all come together as each of the CRISPR field’s startup companies have raised significant financings and geared up for a race to turn the technology into medicine. Editas raised a $120 million round just a few months ago. CRISPR Therapeutics raised $64 million from Celgene and others earlier this year. Intellia grabbed a $70 million round of its own. These companies each have the diverse shareholder base of a public company, or at least one that would consider an IPO, but much scientific work remains before the first CRISPR therapy begins clinical testing. Challenges include figuring out how best to deliver CRISPR therapies, and making sure the molecular scissors that they’re based around make cuts in the right places. It’ll all unfold over the next few years, making for one of the more fascinating sprints in scientific innovation.
“As a company founded on innovative science, we’re excited to begin this collaboration with CRISPR, as it puts us at the forefront of what we believe may be a fundamental change in the future treatment of disease—using gene editing technologies to address the underlying genetic causes of many diseases,” Altshuler said in a statement.