When Angela Yen was growing up, she wanted to be a dancer or a singer — anything but a computer scientist like her father. By the time she got to college at MIT, she’d settled on bioengineering. Until she actually got into a lab. “I messed up all the experiments,” Yen said. “I was so motivated by the project but the physical work didn’t work for me.”
What did work was tackling problems on computers. Her prodigious coding skills carried her through a Ph.D. program in computational biology, and now to a job at Vertex Pharmaceuticals making sure the first CRISPR medicines to be tested in people are safe.
From the earliest days of the CRISPR gene editing revolution, curing genetic disease has been one of its most tantalizing promises. But even as research and investment quickly advanced therapies toward the clinic, crucial questions remained. CRISPR can be unpredictable — making cuts where it shouldn’t, causing random errors, and even deleting whole chromosomes. Any company trying to make a CRISPR-based medicine needed to develop tools for detecting such mistakes.
At Vertex, Yen led that charge, building a computational framework for analyzing potential off-target effects. Her efforts pushed CTX001 — a one-time treatment for sickle cell disease and beta thalassemia being developed jointly by Vertex and CRISPR Therapeutics — into the first human trials.
CTX001 is now the Western drug industry’s most advanced effort to use the Nobel Prize-winning technology to provide functional cures for inherited diseases. In June, the companies reported data showing the first 22 patients to receive CTX001 have been freed from severe pain and the need for regular blood transfusions.
Yen’s methods helped set the industry standard for companies developing CRISPR medicines to treat other diseases. “In retrospect, it’s like ‘of course that’s the right way to do it,’” she said. “But at the time we had to answer a lot of questions about what data to collect and which experiments to do.”
Now as the head of computational genomics for genetic therapies for Vertex, she’s leading a team of scientists figuring out similar questions for other diseases. She still has dance parties, but now they’re mostly with her two young sons.
— Megan Molteni