A new kind of gene editing company has just come out of stealth mode.
Armed with an $87 million Series A funding round announced Monday, Beam Therapeutics aims to develop medicines using a form of CRISPR technology that the company bills as more efficient and precise than those of similar gene-editing platforms.
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The Cambridge, Massachusetts-based firm, which had been in operation for about a year, is touting a “base editing” technology that allows editing of single letters in the genome – A (adenine), G (guanine), T (thymine) and C (cytosine) – rather than the cutting of DNA and RNA that other CRISPR platforms use. Beam has licensed two base editing technologies, developed by co-founder David Liu of Harvard University, which respectively allow C-to-T or G-to-A and A-to-G or T-to-C editing. The company also has licensing agreements with the Massachusetts Institute of Technology’s Broad Institute for co-founder Feng Zhang’s RNA base editing technology and with Editas Medicine for technologies it developed and licensed from Harvard, the Broad Institute and Massachusetts General Hospital.
Venture capital firms ARCH Venture Partners and F-Prime Capital Partners are leading Beam’s Series A raise. The company has hired former Agios Pharmaceuticals executive John Evans as CEO, who is among the 15 employees the company currently has.
The bulk of the funding came from the two VC firms, Evans said in an interview, adding that undisclosed angel investors, family offices and long-term institutional investors are also participating. The funding is expected to last several years, but Beam’s burn rate is expected to accelerate as the company grows and invests, he said. Hiring plans are “a bit of a moving target,” but at least another 10 positions are expected to open. While not providing a timeline, he said the company is growing “quickly.”
Beam is looking at 10-15 disease targets for its technology, but keeping mute about what those are or what specific therapeutic areas the company is looking at in order to avoid prematurely generating anticipation among the public. Apparently, the company has already received emails from patients inquiring about its development programs, Evans said.
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However, he said that when one looks through genetic mutation databases to see which mutations drive disease, about half of them are point mutations stemming from single-letter changes, meaning those are targets that would be fit for base editing. Therapies developed using the platform could be ex vivo – including cell therapies using hematopoietic stem cells or T cells – or in vivo therapies like viral vectors and lipid nanoparticle targets.
The company aims to get a sense of how well its initial efforts are working in those disease targets over the next six to 12 months, followed by animal studies and preclinical work to enable clinical trials, Evans said.
As highlighted by Editas’ $94 million initial public offering two years ago, CRISPR has become a hot topic in gene therapy, a field still dominated by viral vector therapies like Spark Therapeutics’ Luxturna. The latter became the first Food and Drug Administration-approved gene therapy in December, for a rare, inherited form of blindness. In addition to Editas, other CRISPR-focused companies that went public in 2016 include Cambridge-based Intellia Therapeutics and Basel, Switzerland-based Crispr Therapeutics. A report in March by Market Reports Center forecast that the global market for gene therapy would reach $363 million by 2022. However, it defined gene therapy broadly to include Novartis’ Kymriah and Gilead Sciences’ Yescarta, which are CAR-T cell therapies for blood cancers, and Amgen’s Imlygic, an oncolytic viral therapy for melanoma.
But so far, Evans declared Beam does not have any competitors when it comes to base editing. Nevertheless, while declining to say when Beam would move its therapies into animal models, he noted some animal studies have already been published, and he wouldn’t be surprised if other players get involved.
Most recently, a team of researchers in Korea published a study last month in Nature Biotechnology describing A-to-G base editing in mouse models of Duchenne muscular dystrophy. Another study, published in Protein & Cell last August by researchers in China and at Baylor University in Houston, Texas, described C-to-T editing in mouse zygotes.
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