Harvard and MIT launch gene editing company
Group aiming to commercialise Crispr tool secures $87m initial venture capital funding
A US start-up has joined the race to commercialise Crispr, the gene editing tool that is transforming biotechnology. Pioneers of the field at Harvard University and Massachusetts Institute of Technology have launched Beam Therapeutics with $87m initial venture capital funding.
Beam is the first business to use “ base editing ”, a technique developed by the company’s co-founders, David Liu and Feng Zhang, to treat disease. The series A financing is led by F-Prime Capital Partners and Arch Venture Partners.
Crispr previously required scientists to cut DNA, delete or insert genes and then repair the break. The new technology can change individual bases, the chemical “letters” of genetic code, without cutting. It is like moving from scissors-and-paste to editing text with a sharp pencil.
In addition to licensing base editing technology from Prof Liu’s lab at Harvard for human therapeutics, Beam will exploit a separate discovery by Dr Zhang and colleagues at the Broad Institute, a joint research centre of Harvard and MIT.
This extends base editing from DNA, which permanently stores genetic information, to RNA, the related molecule that puts the genome to work by translating its genetic information into protein. RNA editing is like correcting text with temporary ink that soon disappears rather than making indelible marks.
“The two technologies are complementary,” said Prof Liu. “For some acute applications [such as severe inflammation] you might want to make transient changes and then RNA editing would be appropriate. For others, such as correcting an inherited disease, you might want a permanent change through DNA base editing.”
Tens of thousands of “point mutations” — changes in just one of the 3bn DNA letters that make up the human genome — are known to be associated with disease. These could in principle be corrected through base editing. They include neurodegenerative and metabolic diseases, blood disorders and vision or hearing loss.
Beam has launched with 15 full-time researchers in a temporary lab in Cambridge, Massachusetts, and expects to expand quickly when it moves into permanent premises. “It is too early for us to disclose the specific diseases on which we are working or to comment on our timeline to the clinic,” said Prof Liu.
Several other gene editing companies and university groups hope to begin clinical trials in patients this year, working with other versions of Crispr. One is Editas Medicine, set up in 2013 with Dr Zhang and Prof Liu as co-founders; it has a licensing and option agreement with Beam.
The founders of Beam and Editas recently set up yet another gene editing company, Pairwise Plants, which announced $25m series A funding in March. It will apply Crispr to agricultural crops, in collaboration with agrochemical company Monsanto.
Overshadowing Crispr commercialisation is a long-running and still unresolved dispute over patent rights to the underlying technology, which pits the Broad Institute against the University of California, Berkeley.
While declining to comment on the case, Prof Liu said: “The level of collegiality among researchers in the genome editing field is much greater than you might imagine from reports [of the patent dispute].”