- TitleDirector, head of gene-editing technologies
- CompanyBeam Therapeutics
Gaudelli made a scientific breakthrough that is driving forward the booming, fast-moving field of gene editing. Just a few years ago, she was a postdoc in the lab of Harvard’s David Liu, trying to do something that many considered impossible. While Crispr, the revolutionary gene-editing technology, had already begun to shake up science, the technology was not an efficient way to address a number of genetic mutations—particularly, point mutations, where there is a single-letter misspelling in the DNA sequence—that lead to disease.
When fellow postdoc Alexis Komor (see blurb) developed a more precise tool, now known as a base editor, that could replace a single letter of genomic DNA with another—converting a cytosine (C) to a thymine (T)—Gaudelli set her sights on engineering another one. Gaudelli took aim at creating a molecular tool that could change an adenine base (A) to a guanine base (G), a feat that, challengingly, required creating an enzyme not present in nature. The goal was as significant as it was audacious: If successful, Gaudelli would have a technology with the potential to correct roughly half of all point mutations known to cause genetic disease.
It took about two years of painstaking lab work, “evolving” proteins to do things they do not naturally do, but Gaudelli got there. Her and Komor’s base editors became the basis for Beam Therapeutics, the recently IPO’ed biotech that has a market cap of $1.3 billion. Gaudelli is working to advance her discovery there as the company’s head of gene-editing technologies. Though she won’t speculate on when her base-editing technology will be applied in humans, Beam did recently announce its first two development candidates will be aimed at treating sickle cell disease; both use Gaudelli’s technology.