CRISPR

CRISPR (Credit: Ernesto del Aguila III, NHGRI)

A U.S. startup is hoping to commercialise CRISPR by using “base editing.”

The pioneers of the business, known as Beam Therapeutics are from Harvard University and Massachusetts Institute of Technology and launched it with $87 million in initial venture capital funding, writes Financial Times

Beam is the first company 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. But this new technology can change individuals bases, the chemical “letters” of the genetic code, without cutting it. Therefore, this is like moving from scissors-and-paste to editing text with a sharp pencil. 

In addition to this, Beam is looking at exploiting 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. 

“The two technologies are complementary,” explained Professor 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.”

 

Gentler Gene-Editing With MAGESTIC

 

Tens of thousands of “point mutations” – changes in just one of the 3 billion DNA letters that make up the human genome – are known to be associated with a disease. In principle, these could be corrected through base editing, and they include neurodegenerative and metabolic diseases, blood disorders and vision or hearing loss. 

Beam has launched with the support of 15 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 Professor Liu. 

There are a handful of other editing companies and university groups who are hoping to begin clinical trials in patients this year, working with other versions of CRISPR. One of those is Editas Medicine, which was set up in 2013 with Dr. Zhang and Professor Liu as co-founders; it has a licensing and option agreement with Beam. In addition, the founders set up yet another gene editing company, called Pairwise Plants, which announced $25 million series A funding in March. This will begin by applying CRISPR to agriculture crops, in collaboration with agrochemical company Monsanto. 

However, it must be remembered that 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. 

Despite declining to comment on the case, Professor Lie concluded, “The level of collegiality among researchers in the genome editing field is much greater than you might imagine from reports [of the patent dispute].”