A busy, buzzy Vertex finds a high-profile partner in the RNA-drugging world to launch a fresh discovery effort
John Carroll – Editor & Founder
As Vertex began zeroing in on launching its big combo for cystic fibrosis, the oft-lauded biotech started to selectively snap up new technologies that promised to break the therapeutic mold on the way some diseases are treated.
There was the Semma buyout, looking for a cure for diabetes. There was a deal with CRISPR Therapeutics that just produced some landmark data on curing sickle cell disease. They bought Exonics, with a gene editing approach to curing Duchenne muscular dystrophy.
They even did a relatively small deal with Ribometrix in 2019 that indicated a big interest in drugging RNA, correcting errant splicing that triggers disease. But it evidently wasn’t enough on that score.
Today Vertex is announcing a new discovery alliance with Skyhawk Therapeutics, another RNA drugging outfit — working in a field that also includes Arrakis and others — that has attracted the likes of Merck and Genentech, which is broadly interested in oncology and neurodegeneration.
What do we know about the deal?
Not a lot.
Vertex is fronting the pact with $40 million in cash to get things started. And then there’s more than $2 billion in milestones, which indicates the size of its ambitions. What we don’t know — as is often the case with collaborations — is exactly what Vertex is trying to do here.
Skyhawk chief Bill Haney will say what the general focus is: neurological.
And he was also impressed that Vertex’s team under Boston site head Mark Bunnage knew exactly what kind of targets they were interested in. From there, using Skyhawk’s bioinformatics tech, Haney says there was a straightforward path to determining the likelihood of success and creating a partnership.
“They’ve been fantastically fast, clear, fair, insightful,” the CEO tells me. “There’s no slow with a lot of the guys I’ve been dealing with there … We haven’t spent a lot of time because they haven’t needed a lot of time.”
In choosing who to partner with, Haney says it’s important to ally yourself with highly-focused teams. And at Vertex, he’s tracked programs that, from clinical start to BLA finish, took just 3 years.
So those billions of milestones he’s been adding don’t have to be a distant mirage.