The pioneers of CRISPR technology have founded Beam Therapeutics, a new company that will explore base editing, a new, more precise form of gene editing. The company’s research will focus on multiple DNA and RNA base editor platforms developed in the labs of David Liu, PhD, from Harvard University, Feng Zhang, PhD, from the Broad Institute, and J. Keith Joung, MD, PhD, from Massachusetts General Hospital.
Base editing allows scientists to “edit” individual base pairs in the genetic code, rather than “cutting out” a targeted gene, as with traditional CRISPR technologies. With base editors, scientists can precisely target and edit just one base out of billions within the genome, swapping a single base for another and minimizing genetic side effects.
The company’s first license agreement is with Harvard University for developing two base-editing platforms featuring CRISPR/Cas9 technology. In a second agreement with the Broad Institute, Beam Therapeutics is acquiring RNA base-editing technologies, including the RNA editor platform REPAIR, which uses Cas13 linking. Both license agreements provide an initial period of exclusivity for human therapeutic use.
Beam Therapeutics confirmed that it is working on more than a dozen targets (but would not specify which) and is encouraged by the results so far.
Source: Beam Therapeutics press release, May 14, 2018; Fast Company, May 14, 2018.