With $87 Million in Hand, Beam Therapeutics Launches to Fight Disease with CRISPR Base Editing Technology
Published: May 14, 2018 By Alex Keown
Beam Therapeutics, a new precision genetic medicines company, launched this morning with $87 million in a Series A funding round backed by F-Prime Capital Partners and ARCH Venture Partners.
Although Beam did not lay out any specifics regarding its primary disease research, the company said it will focus its research on multiple DNA base editor platforms that were developed in the Harvard University lab of David Liu, as well as on the RNA base editor platform developed by Feng Zhang at the Broad Institute of MIT and Harvard. Beam said it will be the first company to pursue development of new therapies using CRISPR base editing technology.
DNA, Beam said, is made up of billions of nucleobases, or “bases,” each represented by a single letter (A, G, T, C). These letters 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, the company said. The method is more precise than the more common CRISPR programs that cut and edit, or correct, disease-associated DNA in a cell. The base editing technology allows for the rewriting of a mutation in the DNA.
In its coming out announcement, Beam said it will use the different technologies to “generate a broad pipeline of precision genetic medicines that repair disease-causing point mutations, write in protective genetic variations, or modulate the expression or function of disease-causing genes.”
Chief Executive Officer John Evans, most recently head of Corporate Development and Portfolio Leadership at Agios Pharmaceuticals, said base editors are capable of making single-base changes with high efficiency and unprecedented control. Beam, Evans said, has put together the base editing technologies and intends to establish them as a “new therapeutic option for patients with serious diseases.”
Out of the gate Beam Therapeutics has established several licensing agreements. Its first agreement is with Harvard that covers two base editing platforms developed in Liu’s lab. The first is the C base editor, which features Cas9 linked to a cytidine deaminase to deliver programmable C-to-T or G-to-A edits in DNA. The second is the A base editor, which features Cas9 linked to an evolved form of adenosine deaminase capable of editing DNA to deliver programmable A-to-G or T-to-C edits.
The second license is with the Broad Institute for RNA base editing technologies from Zhang’s lab. This includes the RNA editor platform, which features Cas13 linked to an adenosine deaminase to deliver single base A-to-G editing of RNA transcripts, the company said.
Additionally, Beam entered into a third licensing and option agreement with Editas Medicine. Under terms of this agreement Beam secured an exclusive sublicense to patent filings by Harvard for base editing technologies and patent filings by MGH for CRISPR technology. The company also gained an exclusive option for future sublicensing of additional Cas9 patent families and Cpf1 patent families in the field of base editing. In return, Editas Medicine has received an equity stake in Beam and will be eligible for royalties on medicines utilizing the related intellectual property and technologies.
In addition to Evans, Beam’s leadership team includes Chief Scientific Officer Giuseppe Ciaramella, who most recently served as CSO of Moderna Therapeutics’ Infectious Disease division.