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A number of gene-editing companies have joined the public markets in recent years. Beam Therapeutics, which is developing a CRISPR-based technology intended to offer even more precise genomic edits, aims to become the latest one.

Cambridge, MA-based Beam filed its IPO paperwork with securities regulators late Friday. The company set a preliminary $100 million target for the offering. It has applied for a Nasdaq listing under the stock symbol BEAM.

CRISPR-Cas9 gene editing cuts the genome at specific locations in order to remove or add a piece of DNA. But Beam contends theres room to make CRISPR editing even more precise. If you picture the double helix structure of DNA as a ladder, each rung is made up of a base pair, which consists of two bases. Many genomic mutations occur in a single base. Beams technology, called base editing, is being developed to target these single base errors, which are called point mutations.

If existing gene editing approaches are scissors for the genome, our base editors are pencils, erasing and rewriting one letter in the gene, Beam says in its IPO prospectus.

Beam faces plenty of competition in the gene editing space. Other companies using CRISPR-Cas9 technology to develop new therapies include Caribou Biosciences, Editas Medicine (NASDAQ: EDIT), CRISPR Therapeutics (NASDAQ: CRSP), and Intellia Therapeutics (NASDAQ: NTLA). But the ability to edit point mutations could make the Beam technology applicable to a broader range of genetic diseases. The company says point mutations represent 58 percent of all known genetic errors associated with disease.

Beam was founded in 2017. Until now, Beam has kept quiet about which diseases it aims to treat. The companys filing lists 12 programs, including the blood disorders beta thalassemia and sickle cell disease, and the blood cancers acute lymphoblastic leukemia and acute myeloid leukemia. The pipeline also includes potential treatments for liver diseases, as well as disorders of the eye and the central nervous system.

All of Beams programs are preclinical. For most of them, the company says it has demonstrated therapeutically relevant base editing of cells in the lab. Next year, Beam aims to show that it can base edit genes in animals, tests that are slated for next year. If all goes well, Beam says it could start filing for clearance to begin human testing for multiple programs in 2021.

Beam has raised more than $223 million; its most recent financing was a $135 million Series B round in April. The company still has plenty of money in its coffers: As of June 30, Beam reported $126.8 million in cash holdings. The company says it will use the IPO proceeds to continue research and development of its base editing programs. According to the IPO filing, ARCH Venture partners is Beams largest shareholder with a 23 percent stake followed by F-Prime Capital Partners Healthcare Fund, which owns 19.4 percent of the company.

Heres more on the origins of Beam, which is based on research from Harvard University, the Broad Institute, and Massachusetts General Hospital.

Photo by Flickr user marco.savia a Creative Commons license

Frank Vinluan is editor of Xconomy Raleigh-Durham, based in Research Triangle Park. You can reach him at fvinluan [at] xconomy.com

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Beam Therapeutics Preps IPO and Sheds Light on Its Gene-Editing Drugs - Xconomy

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