Three of the founders behind the high profile gene-editing company Editas are taking their scientific prowess to a new venture, launching a startup this week that expands on the pioneering CRISPR tech the entire space is built on.
The new company — called Beam Therapeutics — is stepping out Monday with $87 million in launch money. Investors are backing true trailblazers in gene editing, as Beam’s scientific founders include David Liu, now-serial entrepreneur Feng Zhang, and J Keith Joung. All three are scientific founders of Editas, the CRISPR company launched in 2013 that now garners a $1.7 billion market cap.
Beam is taking a totally new approach to gene editing, hoping to tweak base pairs without actually cutting the strand of DNA or RNA. If CRISPR can be compared to scissors, then Beam’s base editing tech is more like a pencil, Liu tells me, erasing a “bad” base and writing in a good one.
The impetus for Beam’s launch was Liu’s research at Harvard, which is now licensed to the Cambridge startup. The license covers two base editing platforms: a C base editor and an A base editor. The C base one features Cas9 linked to a cytidine deaminase to make C-to-T or G-to-A edits. The A base editor has Cas9 linked to “an evolved form of adenosine deaminase,” which can make A-to-G or T-to-C edits.
Liu’s DNA base editing tech is just part of Beam’s package, though. Zhang, the guy who just months ago helped launched a different gene editing startup (Arbor), has joined forces with Liu, contributing RNA base editing technology from his lab that features Cas13 linked to an adenosine deaminase that can edit A-to-G in RNA transcripts.
On top of licenses to Liu and Zhang’s tech, Beam also has patents from Editas. Beam’s CEO John Evans tells me the deal gives the startup exclusive rights to IP licensed to Editas by Harvard, Broad, and Massachusetts General Hospital, along with certain Editas technology. As part of that deal, Beam has an exclusive sublicense to base editing patents out of Liu’s lab and patents by MGH for CRISPR technology developed in Joung’s lab.
Beam’s approach to gene editing is quite compelling for a number of reasons. First, slicing DNA is a permanent change, and that isn’t always a good idea.
“There are some states of disease that don’t last a lifetime, but are transient,” Liu said. “For those cases, you may not want to address the disease by making a permanent change to the DNA of cells.”
“Erasing and writing in” edits without clipping could be incredibly useful in these cases. Liu said editing mutations in the nucleobases of DNA and RNA has the potential to “reverse a large fraction — perhaps the majority — of point mutations associated with disease.”
But what are the odds that it will work? Liu said he’s happy with early indications. After first publishing work on these base editors back in 2016, dozens of researchers have tried their hand at the technology and saw success in bacteria, fungi, insects, mice, and even human embryos.
Beam wants to stay quiet on which diseases its first targets will tackle but did mention that the company is actively working on 10-15 targets and is “encouraged by early results.” This Series A funding, which was led by F-Prime Capital Partners and ARCH, should take the company “multiple years,” Evans said.
“The length of that runway gives us confidence that we can push forward quickly with lead programs, but also take time to go deep on the science and build a broad pipeline in parallel,” Evans said.
Evans has joined Beam from ARCH, where he was a partner. Although VCs often sit in as temporary CEOs while a startup hunts for a more permanent candidate, Evans said he’s in for the long haul at Beam. While still affiliated with ARCH, Evans is joining Beam full time.
“At Agios, I saw the power of precision medicine to directly correct mutations in leukemia,” Evans said. “That was a powerful experience for me, and I want to do that again. With base editing, I see a real potential to do it again and again.”
Beam has plans to set up an office in the heart of Cambridge’s Central Square neighborhood. It employs 15 people today, and plans on hiring “several more” in the near future.
Top Image: Feng Zhang, David Liu and J Keith Joung. BEAM THERAPEUTICS
The best place to read Endpoints News? In your inbox.
Comprehensive daily news report for those who discover, develop, and market drugs. Join 29,000+ biopharma pros who read Endpoints News by email every day.Free Subscription