Beam Therapeutics will focus on a gene editing technique that does not require cutting.
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) genes are found in certain bacteria and other organisms, and enable these organisms to acquire resistance to invading genetic material through incorporation of genome fragments of the infectious agents. The CRISPR system has been employed for gene editing because it is relatively simple and easy to manipulate.
Editas is one company that has been very successful in leveraging CRISPR technology. Since its founding in 2013, the company has reach a market capitalization of $1.7 billion.
Three of the scientific minds that contributed to the creation of Editas - David Liu, Feng Zhan and J Keith Joung - recently launched a new gene-editing startup named Beam Therapeutics. Investors have contributed $87 million to its launch.
Beam’s gene-editing technology is different from CRISPR. Developed by Liu at Harvard University and now licensed to Beam, the approach can be better compared to erasing DNA/RNA bases (rather than cutting strands) and writing in desirable alternatives. The technology can be used to edit C and A bases. The C editor can change C bases to T bases or G bases to A bases, and consists of the Cas9 protein linked to a cytidine deaminase enzyme. The A editor changes A bases to G bases or T bases to C bases and also consists of the Cas9 protein, but this time it is linked to “an evolved form of adenosine deaminase,” according to Liu.
The new gene-editing technology is attractive because unlike with large, permanent changes to DNA/RNA strands achieved with CRISPR, the changes are focused on individual bases and can address point mutations associated with disease, according to Liu. “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,” he says.
Beam has also licensed RNA editing technology developed by Zhang that uses the Cas13 protein linked to adenosine deaminase that can edit A bases to G bases in RNA transcripts. In addition, the company has obtained exclusive rights to IP licensed to Editas by Harvard, the Broad Institute and Massachusetts General Hospital and certain rights to Editas technology, including an exclusive sublicense to base editing patents out of Liu’s lab and patents by MGH for CRISPR technology developed in Joung’s lab
Initially, Beam will focus on 10-15 targets. Led by F-Prime Capital Partners and ARCH, the Series A funding raised for the launch is expected to take the company through its first several years. The company will establish offices in the Central Square neighborhood of Cambridge.