Harvard University has granted a worldwide license to Beam Therapeutics to develop and commercialize DNA base editing technologies for the treatment of human diseases. Beam raised $87 million in Series A financing to advance the technologies.
Prof. David R. Liu, Professor of Chemistry and Chemical Biology at Harvard University, is the inventor of the base editing technologies and is a cofounder of Beam Therapeutics. He was also a cofounder of one of the original CRISPR companies, Editas Medicine, along with Feng Zhang, an inventor of CRISPR gene editing at Broad Institute, and Keith Joung, a gene editing researcher at Massachusetts General Hospital and Harvard Medical School. Zhang and Joung are cofounders of Beam Therapeutics too.
Existing platforms of genome editing including the CRISPR-Cas9 system works as molecular scissors and makes double-stranded breaks on the DNA, which results in imprecise correction of mutations. On the contrary, the base editing platform developed in Liu’s laboratory use an engineered protein to unzip a targeted portion of the DNA, opening up a small bubble in which to correct the single base, without causing double-stranded breaks in DNA. The base editors can then directly convert the mutated base to the correct base. Liu’s first published base editor can convert C to T and G to A. His second model can convert A to G and T to C.
Base editing represents a powerful tool for addressing a large class of genetic diseases that are more difficult to tackle with other methods of genome editing. According to a database, 33000 single point mutations are associated with disease and the four changes that Liu’s base editors can make could correct 63% of them in principle.
Under the terms of the agreement, Beam Therapeutics will make a multimillion-dollar upfront licensing payment to Harvard university. Additional terms of the deal are confidential.
Prof. Liu commented: “We developed programmable molecular machines that go to a target site of our choosing in the genomic DNA of a cell and directly convert one base to another base without making a double-stranded break in the DNA. For some applications, scissors are the best tools. But if the goal is to simply fix a single-point mutation, base editing is really the best tool.”
Source: Beam Therapeutics Launched to Develop Harvard Base Editing Technology toward Precision Genetic Medicines. Press Release