Beam Therapeutics announced today that it is launching to develop precision genetic medicines that make edits to individual base pairs in the genetic code. Co-founded by scientific pioneers known for advancing CRISPR gene editing, Beam is the first company to pursue development of new therapies using CRISPR base editing technology.

Beam’s research will focus on multiple DNA base editor platforms developed in the lab of David Liu, Ph.D., at Harvard University, as well as on the RNA base editor platform developed by Feng Zhang, Ph.D., at the Broad Institute of MIT and Harvard. DNA is made up of billions of nucleobases, or “bases,” each represented by a single letter (A, G, T, C), which are subsequently encoded in RNA messages for expression by the cell. Base editors are capable of precisely targeting and directly editing just one base out of billions within the genome, without cutting the DNA or RNA.

“Base editors are capable of making single-base changes with high efficiency and unprecedented control,” said Beam CEO John Evans, adding, “Beam has assembled the key technologies in base editing and is dedicated to establishing base editors as a new therapeutic option for patients with serious diseases.”

Beam has licensed access to several cutting-edge base editing platforms. Beam’s first license agreement is with Harvard University for base editing technologies in the field of human therapeutics. This license covers two base editing platforms developed in Liu’s lab in Harvard’s Department of Chemistry and Chemical Biology.

In a second agreement with the Broad Institute, Beam is able to acquire RNA base editing technologies from Zhang’s lab. This includes the RNA editor platform (“REPAIR”), first published in Science in 2017, which features Cas13 linked to an adenosine deaminase to deliver single base A-to-G editing of RNA transcripts.

The Licenses provide Beam with an initial period of exclusivity for human therapeutic use. There is a mechanism to extend a license to others on an individual gene target basis, if the technology is not being actively developed for that target.

Beam has entered into a licensing and option agreement with Editas Medicine for exclusive rights to certain intellectual property licensed to Editas Medicine by Harvard, the Broad Institute, and Massachusetts General Hospital (MGH), as well as to certain Editas Medicine technologies. Under this agreement, Beam has received an exclusive sublicense to patent filings by Harvard for base editing technologies developed in the Liu Lab and patent filings by MGH for CRISPR technology developed in the Joung Lab, as well as an exclusive option for future sublicensing of additional Cas9 patent families and Cpf1 patent families in the field of base editing.