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.

Beam aims to use these 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.

"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."

Leading Portfolio of Base Editing Intellectual Property and Technologies

Beam has assembled the leading portfolio of base editing intellectual property across multiple foundational 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. The first is the C base editor ("BE"), originally published in Nature1 in 2016, 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 ("ABE"), published in Nature2 in 2017, 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.

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.

Both licenses provide an initial period of exclusivity for human therapeutic use, after which time 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.

Finally, 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. 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.

Series A Funding and Founding Team

Beam is launching with up to $87 million in cumulative Series A funding for Beam and an affiliated entity, led by F-Prime Capital Partners and ARCH Venture Partners. CEO John Evans was most recently SVP Corporate Development and Portfolio Leadership at Agios Pharmaceuticals and is also a Venture Partner with ARCH. Giuseppe Ciaramella, Ph.D., most recently CSO of Moderna Therapeutics' Infectious Disease division, will lead research as Chief Scientific Officer. Beam's founders are Liu, Zhang and J. Keith Joung, M.D., Ph.D., a leading researcher at MGH and Harvard Medical School who has developed important technologies for targeted genome editing.

"Our co-founders helped put CRISPR on the map. With Beam, they're coming together again to push forward an exciting and differentiated approach to genome editing," said Dr. Stephen Knight, F-Prime President and Managing Partner.

Robert Nelsen, Managing Director and Co-Founder of ARCH Venture Partners, said, "With this next-generation gene editing technology, world-class team, and significant resources, Beam is uniquely positioned to change how we treat and potentially even prevent diseases."

"Progress in the CRISPR genome editing field continues to accelerate. Beam's base editing technologies create brand new ways of precisely editing the genome to treat patients with devastating diseases," said Kristina Burow, Managing Director of ARCH Venture Partners.

Beam's Board of Directors includes CEO John Evans, investors Kristina Burow, Stephen Knight, and Robert Nelsen, co-founder Feng Zhang, and independent director Mark Fishman, M.D., Professor of Stem Cell and Regenerative Biology at Harvard University and formerly the founding President of the Novartis Institutes of BioMedical Research (NIBR).

About Beam Therapeutics

Beam Therapeutics is developing precision genetic medicines through base editing. Founded by leading scientists in CRISPR gene editing, Beam is pursuing therapies for serious diseases using its proprietary base editing technology, which can make precise edits to single base pairs in DNA and RNA. Headquartered in Cambridge, Massachusetts, the company is backed by F-Prime Capital Partners and ARCH Venture Partners, two of the world's leading biotech venture capital firms. For additional information, visit www.BeamTx.com.

About F-Prime Capital Partners

F-Prime Capital Partners is a global venture capital firm investing in life sciences, health care and technology. Since 1969, F-Prime has worked closely with entrepreneurs and academics to create innovative solutions to some of the world's most significant challenges in health care and technology. For more information, please visit www.fprimecapital.com.

About ARCH Venture Partners

ARCH Venture Partners is an innovator in company creation and early-stage venture capital and invests in seed- and early-stage technology companies. The firm is a recognized leader in commercializing technologies developed at academic institutions, corporate research groups and national laboratories. ARCH creates companies around breakthrough technologies and invests primarily in companies it co-founds with leading scientists and entrepreneurs, bringing innovations in life sciences and physical sciences to market. Over three decades, ARCH has raised more than $3 billion of committed capital through nine venture funds and has financed more than 230 new ventures. The firm has offices in Chicago, San Francisco, Austin, and Seattle. For more information, visit www.archventure.com.