A startup that wants to make treatments that edit single letters in our DNA just raised $87 million

Courtesy Beam Therapeutics

Beam cofounders Feng Zhang, David R. Liu, and J. Keith Joung

• Beam Therapeutics, a new startup co-founded by gene-editing pioneers, just raised $87 million to get its technology that changes single letters in DNA into humans.
• The cutting-edge technology, known as base editing, acts as a molecular pencil, co-founder David Liu told Business Insider. In contrast, CRISPR, another new gene-editing technology acts more like scissors, cutting out larger chunks of DNA on a particular gene.
• The technology is still in early days, though the company said it has 10-15 programs with early data.

A startup co-founded by gene editing pioneers wants to use technology that makes changes to single letters of DNA to treat diseases.

Called Beam Therapeutics, the company just raised $87 million in a series A from investors including F-Prime Capital Partners and ARCH Venture Partners. The cutting-edge technology it's putting to work is known as base editing, and it acts as a molecular pencil, co-founder David Liu told Business Insider.

Our bodies are made up of 3 billion base pairs, the As, Cs, Ts, and Gs that make us who we are as individuals.

Of the roughly 60,000 genetic diseases that exist, roughly half of them can be attributed to point mutations, Liu said. These point mutations happen when a single base pair is altered.

What Beam wants to do is use base editing as a pencil (erasing one letter and putting in another) to correct just that one letter - swapping a C to a T or a G to an A. Beam's licensed technology that explores how that change works in the person's DNA, or in the RNA that then encodes a particular protein.

Liu, who also co-founded CRISPR gene-editing company Editas and a professor of chemistry and chemical biology at Harvard University, said he sees base editing as complementary to the work that's being done with CRISPR, which he thinks of more as molecular scissors.

"There are thousands of human genetic diseases that society would love to address and there's no one technology that's going to be able to address them all," Liu said. "I think we all have a responsibility to try to develop as many of them as we can to cover as many conditions that are treatable as possible."

The technology on the DNA base-editing and RNA base-editing fronts are both relatively new. Liu and his lab first published information DNA base editing in the journal Nature in 2016. MIT biologist Feng Zhang RNA base-editing tool

As the researchers were working in their own labs to better understand base editing, they saw that other labs were also testing the technology out and validating it. Eventually, Liu said, starting a company emerged as the best option to advance the technology and ideally one day start benefiting people.

The technology is still in early days, though the company said it has 10-15 programs with early data. Liu said the criteria the company's using to pick where to start include:

• Making sure that in the particular disease, changing a single base can reverse the disease.
• Determining whether the the DNA or RNA sequence is a good fit for a base editor, so it can target and make the right kind of edit.
• The therapy can get to the tissue in the body that's being treated. For example, some gene-editing companies that use CRISPR are starting with treatments to eye or liver conditions, or blood disorders.