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RNA

Movers And Shakers

Jennifer Petter wants to interfere with RNA to discover new drug targets

This entrepreneurial chemist is blazing new paths in biotech

by Neil Savage, special to C&EN
April 8, 2022 | A version of this story appeared in Volume 100, Issue 12

 

A photo of Jennifer Petter.
Credit: Vanessa Leroy Photography
Jennifer Petter

Jennifer Petter was trying to decide what her next career move should be when she sat in on a 2015 conference session about small molecules interacting with folded RNA. The talk described how such molecules could interfere with the folding of RNA before it even produced a protein, possibly providing a new way to find drug targets for previously undruggable diseases.

Vitals

Hometown: Stow, Massachusetts

Education: AB, Dartmouth College, 1978; PhD, Duke University, 1983

Current position: Founder and chief innovation officer, Arrakis Therapeutics

LGBTQ+ identity: Transgender woman

Favorite lab tool: Lab tool? Oh, honey, if I walk into the lab, someone rushes up and asks me, “Can I help you? Don’t touch that! Are you looking for somebody?”

Precious pets: I have 17 dogs, 16 of them collies. I breed them and show them.

“I decided instantly that I wanted to do that because it seemed like an interesting problem,” says Petter, who soon thereafter founded Arrakis Therapeutics, where she is chief innovation officer. What made the problem interesting to her was, in large part, how difficult it seemed. “You really want to be out there solving problems that other people have not solved and blaze new trails,” she says. “Well, this was as new as they come, honey.”

Petter moved into industry in 1991 after 7 years as an assistant professor at the University of Pittsburgh. In 2012 she was at Avila Therapeutics when Celgene bought the company and made her vice president of chemistry.

Although she enjoyed the job, it involved a lot of travel, and eventually she decided she’d rather do something that kept her close to home with her teenage children. That led to her founding Arrakis, named for the planet in Dune with the giant sandworms. “Arrakis is the planet that produces precisely one thing,” she says, explaining the name choice. Arrakis is mined for spice, a drug that’s orally active and can penetrate the central nervous system—both qualities Petter hopes her company’s RNA-degrading therapeutics will share.

Traditional small-molecule drugs work by binding to a protein involved in a disease process and altering that protein’s activity. Arrakis, on the other hand, wants to find molecules that will stop the production of a target protein by binding to its RNA and locking its structure in place. RNA often has a folded structure that must unfold for messenger RNA to be translated into a protein. “RNA is upstream of all biology,” Petter says. “So insofar as you can solve this problem, you have the potential to address pretty much any disease.”

For instance, the company is studying Myc, a protein that plays an important role in cancer. Scientists have not yet developed any drug that can effectively target it. “It’s a classic undruggable, but we sort of hit the reset button,” Petter says. “We’re going after the RNA that makes the Myc rather than the Myc itself.”

Arrakis has not yet proved that its small molecules can target and lock up RNA sequences. And when it does, it will need to show that these molecules can significantly affect disease. But Petter says she’s encouraged by the company’s internal data and hopes to have a candidate molecule for development by the end of this year.

You really want to be out there solving problems that other people have not solved and blaze new trails. Well, this was as new as they come, honey.

Other pharmaceutical companies are also encouraged. In January, Amgen announced it would pay the company $75 million to research targeted RNA degraders for five diseases yet to be selected. Last year Arrakis signed a similar development collaboration agreement with Roche, for $190 million.

Michael Gilman, CEO of Arrakis, says he was skeptical when Petter first came to him with the idea for the company. “At the time, the idea that you could identify drug-like small molecules that bind RNA seemed like a crazy flyer—that’s certainly what most investors told us,” he says. “But I knew from my experience with Jen—I’d known her for over 15 years at that point—that if she thought it could be done, it could.”

The pair had been friends for a long time. In March 2018, as the company was preparing for its B round of fundraising, Petter had dinner with Gilman and told him her news. Though at that point she presented as a man, she’d been on hormone replacement therapy for over a year and would soon be publicly transitioning. That dinner with Gilman was followed by a board meeting in April and a party in May, when she came out to the entire company as transgender. She’d told her three children a few months earlier. By June, she was showing up to work as Jennifer.

Her decision to come out was triggered by her wife’s 2011 diagnosis of breast cancer. Her wife ultimately survived, but the “near-death experience” changed Petter’s thinking. “This event affected me deeply, in ways that took time to understand, most notably realizing that none of us is granted forever,” she says.

Transitioning while fundraising was challenging. She felt anxious in the moments before stepping into meetings with potential investors who thought they were going to be seeing a man. “There are a number of rooms you’re going to walk in, and you just have to suck it up and be who you are, present your slides, and present the science and answer the questions,” she says. “Once you’re there, it’s like, ‘Well, you pulled the trigger, honey. We’re doing this.’ ”

The B round was successful, raising $75 million.

And overall, Petter says colleagues have been supportive of her transition. “It would be difficult to imagine how it could have gone better,” she says. “And for that, I’m deeply appreciative.”

The fact that there are few openly trans people at senior levels in science and industry makes the process of coming out somewhat challenging, Petter says. “The pathway is sufficiently unknown that you don’t know which of your anxieties are real and true and worth worrying about, and which ones are nothing to get worked up about,” she says. She has no particular advice for younger LGBTQ+ people in the sciences because everyone’s path is so different. “I try to take people where they’re at, hear what their stories are,” she says. “To the extent that my story is of any help to them, I’m delighted to tell it.”

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