Vertex adds a new gene editing partner, paying Obsidian $75M for controllable CRISPR system
When Paul Wotton became CEO of Obsidian Therapeutics two years ago, he asked his team to explore where they could apply the company’s technology beyond cancer.
Launched by Michael Gilman in 2017, the GV-backed company had been working on a way of controlling cell therapies after they’re administered to a patient, allowing doctors to modulate the potency of CAR-T and other drugs as you might volume on a television set. Wotton wanted to them look beyond cancer, into areas such as gene editing.
“Gene editing was particularly attractive because it’s a platform approach, where we can really make a difference,” Wotton told Endpoints News. “And it gave us a lot of flexibility with respect to growing the company through business development, while we focused on our own programs.”
That search has now paid off, to the tune of up to $75 million over the next 4 years and $1.3 billion in long-term milestones. On Thursday, Obsidian announced that they struck a deal with Vertex to use the biotech’s technology’s to develop controllable gene editing therapies. Although the partners didn’t disclose what diseases they’re going after, Wotton said they hoped to have a candidate in the clinic in 3 to 4 years.
For Vertex, the deal represents another investment in gene editing. The Cambridge drugmaker, best known for its cystic fibrosis pills, first invested in the field in 2015 when they struck a long-term collaboration with CRISPR Therapeutics, but it’s become a greater focus as they looked to push into new disease areas.
Two years ago, they bought out the Duchenne muscular dystrophy gene editing biotech Exonics, and they are now building a new facility dedicated solely to cell and gene therapy. Earlier this week, they spent $900 million to take an additional 10% stake and operational control of the sickle cell therapy they developed with CRISPR.
In a statement, Vertex CSO David Altshuler noted the deal also plays into the company’s experience with small molecules, like their CF drugs. Obsidian’s technology uses approved small molecule drugs as the “on/off” switch for cell or gene therapies in the body.
“This collaboration with Obsidian builds upon and will expand Vertex’s leadership in small molecule and genetic therapies, and we’re excited to partner with the team at Obsidian to explore the capabilities of their technology,” Altshuler said. “The ability to tune gene-editing activity to a specific level is an important innovation that has the potential to address several serious diseases.”
Wotton said Obsidian has figured out how to control the Cas9 protein that CRISPR systems use to cut DNA. Essentially, they add a peptide tag on top of Cas9. The protein is only active when doctors give a particular small molecule that binds to that tag. Withdraw the small molecule, and Cas9 stops cutting.
“It’s like a rheostat,” Wotton said of Obsidian’s tech. “It allows you to dial up or dial down the activity of a specific protein.”
In cancer, Obsidian is trying to give doctors a tool to turn down immune-boosting therapies that can sometimes attack healthy tissue in addition to tumors.
With Vertex, the companies will try to develop a system that boosts Cas9’s DNA-cutting abilities when they’re first administered, but then turn down its activity once the edit is made.