Gene Editing, People

Merck R&D chief Roger Perlmutter offers some heavyweight R&D glam to a little upstart biotech out to cure Duchenne MD

Back when Roger Perlmutter jumped into a leading role as R&D chief at Merck, one of the first things he did was bow out of the round of board positions he had built up after departing Amgen.

It didn’t make any sense to divide his time, he tells me, until things quieted down at the pharma giant, which has been spearheading the revolution in PD-1/L1 checkpoint inhibitors with hundreds of ongoing studies involving Keytruda.

Now, 5 years later, he’s making an ultra rare exception, joining the board of a small, Cambridge, MA-based upstart that is working on taking the advances that Eric Olson at UT Southwestern made and pushing into the clinic with a CRISPR/Cas9 approach to gene editing that could repair exon mutations that trigger lethal cases of Duchenne MD.

“It looked like a really great setting to use that gene editing approach,” Perlmutter tells me about his new board role. “I never thought [CRISPR/Cas9] could be applied in a large scale clinical setting, so it’s not the sort of thing I would do here at Merck.”

David Goeddel

Over the years, Perlmutter has occasionally focused favorable attention on certain biotechs, often based on his relationship with 1 or 2 of the people at the company and his interest in the technology they were working with. In this case, Exonics chairman and the man who just arranged a $40 million launch round, Dave Goeddel from The Column Group — where Perlmutter is a science partner — made the overture.

And Perlmutter counts himself as a longtime admirer of the principal scientist behind Exonics’ preclinical program.

“Eric Olson is just a terrific molecular biologist,” says Perlmutter effusively.

“I must say that I had to sell many people over time on what I thought might be a challenging decision to make based on their busy schedules,” says Exonics CEO John Ripple. Perlmutter, though, “was instantly interested.”

John Ripple

It’s not lost on anyone at Exonics — one of this year’s Endpoints 11 biotechs — that Perlmutter’s unusual presence on the board offers a high-profile validation of their early stage work. And that will attract more interest as they wind their way to the clinic — a twisting and dangerous path under the best of circumstances.

For Perlmutter, it’s a chance to apply some of his knowledge in translational science, helping guide the company to the clinic and its first rendezvous with a patient. And first up he’ll be helping out with identifying recruits for the team at Exonics.

“I think all companies irrespective of size face the same challenge of identifying places where very high quality scientific thinking can be translated into medicines that make a real difference,” says Perlmutter. “It’s still the case. It all comes down to project champions and their scientific insights, and that’s the same problem everywhere.”

“It comes down to picking targets and pursuing them assiduously and with great clarity.

“That’s true for everybody.”


Image: Roger Perlmutter. Merck


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