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Vertex doubles down on gene editing in $900 million investment with CRISPR Therapeutics

Deal will help the Boston biotech diversify beyond its core suite of cystic fibrosis treatments

A sign at the Vertex Pharmaceuticals headquarters in the Seaport.
A sign at the Vertex Pharmaceuticals headquarters in the Seaport.Nic Antaya for The Boston Globe/file

Vertex Pharmaceuticals made a big investment in 2015 in a then-unproven gene editing technology, with the hopes of finding new ways to treat genetic disorders.

Now, with proof of this technology’s effectiveness in hand, Vertex is doubling down: The Boston-based biotech on Tuesday announced it will pay at least another $900 million to strengthen a partnership it inked six years ago with CRISPR Therapeutics. The goal: to develop, make, and commercialize a gene editing therapy that could cure sickle cell disease and a similar inherited blood disorder known as beta thalassemia.

Jeff Leiden, executive chairman at Vertex, said the company’s recent clinical studies indicate this is the first time that the gene editing technology has been shown to cure a disease. But he expects it will not be the last.

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“Once in every generation, there’s a huge medical breakthrough, something that changes the world of medicine,” Leiden said. “Gene editing is the next one.”

Vertex initially invested $105 million for a 50 percent stake in a drug development venture with CRISPR Therapeutics, whose primary operation is in Cambridge, although it remains headquartered in Switzerland. Under the original terms of the deal, CRISPR would have led the commercialization in the US of any treatments that emerged from the partnership. Now, Vertex will take over that role for the sickle-cell treatment, on a global basis — a decision made in large part because the company already has a large-scale manufacturing and sales network in place.

Vertex now will be responsible for 60 percent of the costs, and receive 60 percent of the profits from future sales of the drug, which is still only known by its clinical-trial name, CTX001. In return, Vertex will pay CRISPR Therapeutics $900 million upfront, with the potential for another $200 million payment if and when the drug achieves its first regulatory approval.

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Shares in CRISPR Therapeutics rose nearly 6 percent, to $121.67, after the news was announced on Tuesday; Vertex’s stock remained essentially unchanged, finishing the day at $220.16 a share.

The venture with CRISPR Therapeutics is one of several steps Vertex is taking to diversify beyond its core suite of cystic fibrosis treatments, which were responsible for essentially all of the company’s $6.2 billion in revenues last year. Several other local biotechs, including Intellia Therapeutics and Beam Therapeutics, are also pursuing gene-editing treatments for sickle cell disease.

Sickle cell disease and beta thalassemia interfere with how hemoglobin carries oxygen in the body, and usually considerably shorten the lives of patients who have them. Sickle cell disease is most often found in the US among those of African American descent. It affects roughly 100,000 Americans, according to the CDC.

The gene-editing technology used in the Vertex trials is referred to as CRISPR/Cas9. The treatment process essentially involves removing blood stem cells from a patient, using an enzyme to edit the DNA of those cells in a lab, and then reinserting the cells with edited genes back in the patient through a bone marrow transplant. The one-time treatment has been proven effective in 10 patients so far in the clinical trials. Vertex has treated more than 30, but only has data on the 10 at this point.

Leiden said this treatment represents a multibillion-dollar opportunity for Vertex. The company expects to submit the trial results to regulators in the US and Europe for approval within the next two years, with data from as many as 90 patients. The drug has the potential to help tens of thousands of patients live longer lives, with few if any of the painful symptoms or hospital visits normally associated with these disorders. (The price has not yet been determined.)

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“This has the potential to be a game changer,” said Joan Reede, the dean for diversity and community partnership at Harvard Medical School.

Reede said she recalls the pain of seeing patients suffer from sickle cell disease while she was a medical resident at Johns Hopkins University in Baltimore, more than 30 years ago, when the average life span of the patients was much shorter. Also, she had a sister-in-law who died from the disease. She said she hopes other major drug companies will follow Vertex’s lead.

“To me, it is wonderful to see biotechnology and researchers coming together to say, ‘These types of diseases that disproportionately impact some of our population need to be addressed,’ ” Reede said. “When we think about equity, we think about equal access to leading a fulfilled life. For me, this is huge that Vertex is taking this on.”

To Leiden, who also chairs the Massachusetts Competitive Partnership coalition of chief executives, the success of the Vertex-CRISPR Therapeutics relationship underscores the value of Greater Boston’s life sciences cluster — having so many bright minds concentrated in such a tight geographic area, trying to solve huge medical problems.

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“This is the perfect story of Boston innovation, this is the one plus one equals three,” Leiden said. “It’s why we have to really nurture this ecosystem. There’s so much innovation that comes out of the density of these companies.”


Jon Chesto can be reached at jon.chesto@globe.com. Follow him on Twitter @jonchesto.