Big Impact in Bio: Funding the Translation From Bench to Bedside at the American Academy

At a meeting of the American Academy of Neurology last year, Megan Krench saw something remarkable: Children, previously unable to move their head or sit up, were turning their heads to look at their parents, sitting up, and even walking. It was a video of a company’s clinical trial for a gene therapy to treat a devastating childhood neuromuscular disorder, spinal muscular atrophy. Infants with this disease typically never reach any developmental milestones, and most die by age two.

“It's a truly heartbreaking disease, and for as long as it's been around, parents have been told there is nothing that anyone can do; they have no choice but to watch as their baby's health declines, watch their child be put on a respirator, and then ultimately, lose their child far too early,” Krench explains. But at this conference, Krench saw a glimmer of a new future for these children: In one clip from the clinical trial, a toddler ran down the hall with tiny briefcase and stood on his tiptoes to push the elevator button. “The entire presentation hall – thousands of physicians – broke into applause, a minute-long standing ovation. I've never seen anything like that.” And the best part, she says, is that in working for a biotech investment fund she gets to see breakthroughs like this all the time.

An Amgen Scholars alumna (’08), Krench never imagined she’d be working for an investment fund. “When I started my Ph.D., I thought I would contribute to the process through bench research, but after a few years, I realized academic research was further away from the patients than I wanted to be,” she says. When she landed an internship at a boutique biotech consulting firm, however, she began to see new possibilities. “It opened my eyes to all the amazing ideas these biotechs were bringing forward at a breakneck pace,” Krench recalls. “There was this tangible electricity to the space, the feeling that you were right on the edge of something big, something that could really make a difference to patients' lives in the timescale of months instead of the 5- to 10-year timelines I was accustomed to thinking about from my bench research.” 

At the investment firm where she now works, RA Capital Management, Krench evaluates data from companies who are developing novel therapeutics to determine if they have the evidence to support their ideas. This due diligence follows well from Ph.D. work, answering questions such as: “Does the mechanism make sense,” “Are the correct controls included,” “Are the data reproducible,” “Did they use the correct statistical methods,” “Are there baseline imbalances or other factors that could affect interpretation of the results,” and “Are these experiments the right ones to answer the questions at hand?”

“I would never have guessed so much deep scientific diligence goes into investment decision-making,” Krench says. “There are an incredible number of biotech companies seeking capital in both the public and private market, so investment funds need to be able to dive in really deep to decide which companies have data that supports a path to successful drug development.” 

Krench credits the Amgen Scholars Program with her current success in a couple ways. First, the Program introduced her to what eventually became my Ph.D. program at MIT: “Amgen Scholars was how I first became exposed to the incredibly stimulating, diverse, one-of-kind environment that is MIT.” She got her Ph.D. at MIT in neuroscience in 2016, eight years after her Amgen Scholars summer there.

She also received pivotal support from the Amgen Scholars Program through the Alumni Travel Award, which she used to present her Ph.D. thesis research during the student symposium of a Gordon Research Conference, a small event in Italy that year. “The connections I made at that conference have turned out to be incredibly useful,” she says both for early and her career and now. “Those are the premier scientists driving forward innovation in this disease space, helping me keep up with the latest developments in the field.” 

Krench’s own path has further reinforced a valuable lesson: Try everything once. “I really did not think I was going to like the biotech consulting internship. I pictured it being boring and full of spreadsheets and financial stuff, though I had no idea what that even meant,” she says. “But it turned out to be this awesome, dynamic internship where I was able to be exposed to all these interesting processes that I never knew anything about.”

In her daily work, Krench lives for the "a ha!" moments when she finds the best way to communicate a deeply complex idea. “It could be through a graphic, a color-coded table, or a great analogy,” she says. “At the end of the day, I could learn all the information that's ever been published about a topic, but if I can't explain my findings to my colleagues and transfer my knowledge to them, then it's useless.”

To learn more about the Amgen Scholars Program, please visit AmgenScholars.com and check out the #AmgenScholars hashtag on Twitter. Follow @AmgenFoundation to stay up to date with all STEM-related news from the Amgen Foundation.