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Latest CRISPR start-up comes out of stealth mode with $87M Series A


By Jesse Schwartz
Published: May 16th, 2018

Feng Zhang’s latest CRISPR company, Beam Therapeutics, officially came out of stealth this week with $87 million in series A funding. Its mission is to develop “precision genetic medicines” that use CRISPR technology to edit just one nucleotide base, or “letter,” in the genome at a time. The company is based on patented technology developed at the Broad Institute, Harvard, and Massachusetts General Hospital, as well as Zhang’s earlier start-up, Editas Medicine. 

While traditional CRISPR is often compared to making a scissor cut in DNA, Beam’s base-editing technology is more like a pencil, says CEO John Evans. When it reaches its target, it finds a letter, erases it and rewrites it as a different letter without disrupting the sequence of other letters around it, he explained. This makes it particularly useful for correcting point mutations, where a single base in a sequence is changed, inserted or deleted.

Potentially, the Beam technology could be used to treat a wide range of genetic illnesses. “Over half of the genetic changes known to drive disease in clinical databases are point mutations, misspellings in the genome,” Evans said.

The company has not yet disclosed its areas of focus, but Evans laid out three criteria for targets it will pursue. “Do we know enough about the biology of the disease to believe that changing a single base will cure it? Is the genetic sequence a good fit for the base editors to land there? And can we deliver the editor into the right tissue to cure the disease?” Evans said. 

Evans said Editas and Beam are offering two different approaches that should not compete against each other. “Our job at Beam will be to find the diseases for which base editing will be the best solution. Equally, there will be diseases where [traditional CRISPR] will be best,” he said.  

Beam will use its Series A funding to advance multiple base-editing platforms across a broad pipeline, while also testing out different delivery methods, Evans said. The financing, led by F-Prime Capital Partners and ARCH Venture Partners, will bankroll Beam for several years of development at a “pretty accelerated pace,” he added. 

Along with its launch and Series A, the company also announced three license agreements — with Harvard, the Broad, and Editas Medicine. The first covers base-editing platforms developed in the lab of David Liu, one of Beam’s scientific founders. The second agreement covers RNA-targeted base editors that make reversible changes to DNA. And the Editas license gives Beam the rights to certain IP in a sublicense from Harvard, the Broad, and Mass General, as well as some of Editas’ technologies. In return, Editas picked up a stake in Beam and will be eligible for royalties on treatments that use its technology. 

The Harvard agreement sets diligence milestones and stipulates mechanisms by which a third party can propose to develop a therapeutic product, either in partnership with Beam, via a sublicense, or through a separate agreement with the university if the product is not of interest to Beam. The university, as it does in all agreement, retains the right continue using the IP for educational and not-for-profit research purposes. The IP is also available for licensing on a non-exclusive basis for industrial research purposes.

The deal nets Harvard a multimillion-dollar upfront payment from Beam, but specifics and other terms are being held confidential.

“Our goal is to see this innovation develop into transformative treatments for the widest possible range of human diseases,” said Vivian Berlin, director of business development in Harvard’s Office of Technology Development . “Licensing the commercial rights to a new start-up ensures a rapid mobilization of resources to fully develop and exploit the technology in this field.”

Added Isaac T. Kohlberg, senior associate provost and chief technology development officer at Harvard. “The opportunity here is not only to tackle disease but perhaps to fundamentally change the practice of medicine.”

Sources: Fierce Biotech and EurekAlert!

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