The 20 under 40 : Inside the next generation of biopharma leaders
Inside the next generation of biopharma leaders – Endpoints News
“Each generation needs a new music,” Francis Crick wrote in 1988, reflecting back on his landmark discovery. Crick was 35, then, in 1953, when he began working with a 23-year-old named James Watson, and 37 when the pair unveiled the double helix. Rosalind Franklin, whose diffraction work undergirded their metal model, was 32.
The model would become the score for a new era in biology, one devoted to cracking the basic structures turning inside life. Subsequent years would bring new conductors and new rhythms: Robert Swanson, 29 when he convinced a 39-year-old Herb Boyer to build a company off his work and call it Genentech; Phillip Sharp, 29 when he discovered RNA splicing and 34 when he co-founded Biogen; Frances Arnold, 36 when she pioneered directed evolution; Feng Zhang, 31 when he published his CRISPR paper.
You can think of the list below as a playlist of 20 songs. We’ve brought you 20 researchers and executives thinking through ideas that may succeed or sputter, but will certainly help define the soundtrack for the next 20 years of biopharma: computer scientists cracking gene networks and designing biological search engines, venture capitalists burrowing research pipelines into universities, and microbiologists creating antibiotics ‘crapsules’ from feces.
KATHLEEN MCCARTHY – Skyhawk Therapeutics – 35
A scientist corrals top talent in her entrepreneurial ride
U-2 Dragon Lady, one of the conference rooms at Skyhawk Therapeutics’ office in Waltham, MA, is named after both the American single-jet engine, high altitude reconnaissance aircraft and founder Kathleen McCarthy.
Speaking from the Starfire room, McCarthy told Endpoints News that it all arose from serendipity: They’d named the company Skyhawk because it sounded like a majestic soaring bird, only to realize it’s also a Vietnam era plane that Senator John McCain used to fly. So when it came time to name the conference rooms — a team-building exercise — fighter jets became the obvious theme.
From the beginning of her career, McCarthy has been drawn to small organizations that resemble a light attack aircraft trying to have a big impact.
Long before she built Skyhawk to its current size of over 70 — even before the company was called Rare Genetx and consisted of just herself working out of a basement in the Cambridge BioLabs — as an undergrad at Wellesley, McCarthy started a non-profit to nurse kids in a poor neighborhood in the Dominican Republic. Through that project she met Bill Haney, a neighbor of a doctor she was working with, who gave them the first grant to start a preschool and would later become Skyhawk’s angel investor and co-founder. It also reaffirmed her interest in working at the intersection of applied science and public health.
After completing a Fulbright program in Switzerland, McCarthy landed her first job in the Spinal Muscular Atrophy Foundation as it was just beginning mouse model work for a small molecule drug discovered by partners at PTC Therapeutics. As one of five employees, she learned to multitask different types of projects, organizing meetings with experts, analyzing data, designing new experiments and even making presentation slides and evaluating budgets.
“They kind of promised me that no day would be the same, and that’s actually what happened,” she said.
All of that diverse work fed into one ultimate goal: to advance risdiplam, a small molecule drug invented by Sergey Paushkin, who moved from PTC to the SMA Foundation (and is now VP of biology at Skyhawk).
So when Roche got interested in the potential of risdiplam to increase the levels of the essential SMN protein and improve survival, McCarthy was recruited to the pharma giant’s rare disease unit as a non-clinical pharmacologist. Aside from the SMA drug, which is now on the cusp of an OK, she also contributed to several other projects.
As her team began to back-engineer how risdiplam worked around 2014 and 2015, though, they came up with some surprising findings. Chief among them was the realization that the molecule was directly binding to RNA — or more specifically, modifying how pre-mRNA is spliced, thereby spurring the production of functional SMN protein.
“That was enough for me to say you know, I think I have enough information,” she said. “I definitely don’t have the full story, I don’t know how broadly applicable this is, but my hunch was like okay, this is really exciting, super-novel, I’m gonna go try and raise some money to start a company.”
She was advised to get going early, and she did. Balancing the science (by contracting out to CROs) and business (hiring IP lawyers to start grabbing patents) sides of things wasn’t as challenging as figuring out how much they could raise from investors.
Her sister, Maura, who used to work in venture capital before joining Skyhawk’s corporate development team, served as a guidepost at those moments.
“When we got rejected, which we got rejected a lot, she would say, you know, it doesn’t matter, you’re gonna have to do 40 of these, or 50 of these, to eventually raise the money,” she recalled. Then there was the science backing her up: “I knew I had some really exciting, unique results. I knew other people did not have or know, or weren’t targeting RNA the same way I was thinking about it.”
Having an extensive network helped, too, with Haney and Mike Luzzio — a Biogen vet and Skyhawk’s first employee — each bringing in different contacts. Drawing from her liaisons dating back to the SMA Foundation days, McCarthy also got leading RNA researchers Adrian Krainer and Fred Allain early on a star-studded scientific advisory board.
Skyhawk came of age at a time enough rival biotechs are springing up, from Arrakis to Ribometrix, to convince VCs that the moment for RNA-targeting small molecule drugs has arrived, she added.
The constellation has enabled Skyhawk to follow up its first deal, a $60 million upfront arrangement with Celgene, with a slate of new pacts featuring GSK, Merck, Takeda and Biogen.
“The overall question is how broadly do these things apply. And then if it applies everywhere, how do you effectively interrogate each type of target and figure out what the list of targets are related to those,” she said. “So that’s a really fun discovery question.”
It’s a question that the scientist in McCarthy is excited to find the answer to. And the public health advocate in her is itching to see the benefits reach smaller and smaller patient populations, down to n of 1, suffering from single-point genetic mutations. It’s a high-in-the-sky dream, she reckoned, but she’s ready to build the fleet needed to soar to those heights.