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A busy, buzzy Vertex finds a high-profile partner in the RNA-drugging world to launch a fresh ...

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.

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Maura McCarthy


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