Beam was formed five years ago around research from the laboratory of Harvard University's David Liu that showed a way to edit genes more precisely. Unlike the first generation of CRISPR-based gene editing, which cuts both strands of DNA, base editing is designed to change a single DNA "letter" into another without that double-stranded break.
Its potential, like CRISPR's, is vast. The technology could be used to correct disease-causing mutations, to silence genes or activate them, and in theory carries lower risk of causing unwanted genetic changes. Beam, which recently began the first clinical trial involving base editing, plans to develop treatments for blood diseases like sickle cell and beta thalassemia as well as for rare diseases of the liver and eye.
One of the steepest challenges facing Beam, along with other gene editing developers, is developing reliable ways to get the nucleic acid and proteins that make up its editors into cells. For editing inside the body, Beam encodes the editing machinery in mRNA, which is then wrapped inside a fatty shell known as a lipid nanoparticle.
Those two components — an mRNA sequence and a lipid nanoparticle — are the core of mRNA vaccines like the one developed by Pfizer and its partner BioNTech for COVID-19.
"I think Pfizer was looking at the capabilities they had built in lipid nanoparticles and mRNA and saying, 'Well, what are we going to do with these capabilities now that we've built them?'" said Evans.
