Gene therapy, make way for gene “editing.”
Today, three of Boston’s biotech startup creators—Polaris Partners, Third Rock Ventures, and Flagship Ventures, have banded together to create a new, Cambridge, MA-based startup called Editas Medicine.
It’s a sizable bet. The three, with the help of Partners Innovation Fund, have plunked down a $43 million Series A into the new startup, which has ambitious plans to create an entirely new class of drugs based on what it calls “gene editing.” The idea is similar, yet different, from gene therapy: Editas’ goal is to essentially target disorders caused by a singular genetic defect, and using a proprietary in-house technology, create a drug that can “edit” out the abnormality so that it becomes a normal, functional gene—potentially, in a single treatment.
“I would think of this as really the potential for a whole new class of therapeutics,” says Kevin Bitterman, a principal at Polaris and Editas’ interim CEO. “We can go in and, without really any limitation since it’s a programmable system—we can target any gene in the genome and theoretically fix a mistake, remove a gene, or modulate expression in a very targeted way.”
Editas, which has been operating in stealth mode for a few months, is being tight-lipped so far about how it wants to apply this. Bitterman wouldn’t disclose, for instance, what molecular targets the company plans to initially focus on, which types of diseases it wants to go after, how much of the $43 million Editas has access to off the bat, or even what kind of intellectual property Editas is amassing to protect itself from competitors. But it’s been toiling away behind the scenes developing a platform based on the work of five of the most prominent researchers in the field: Feng Zhang, of the Broad Institute of MIT and Harvard; Harvard University genomics expert George Church; Jennifer Doudna, a Howard Hughes Medical Institute investigator and a professor at the University of California in Berkeley; David Liu, another Howard Hughes Investigator and Harvard professor; and Keith Joung, a pathologist at Massachusetts General Hospital as well as a Harvard professor.
It’s also assembled a board of Third Rock (Alexis Borisy), Flagship (Douglas Cole), and Polaris (Terry McGuire) members, and three executives to lead the effort initially: Bitterman, Third Rock partner Lou Tartaglia (interim chief scientific officer), and Alexandra “Sandra” Glucksmann (interim chief operating officer), a former co-founder of Cerulean Pharma, another Polaris portfolio company.
Editas’s big plan is to cure genetic diseases by engineering a process that mimics the adaptive immune system of bacteria. It’s long been known, for instance, that two pieces of bacterial cellular machinery—CRISPR (clustered, regularly interspaced palindromic repeats) and an enzyme called Cas9, or CRISPR-associated protein 9—help bacteria form an adaptive immune system by essentially memorizing the appearance of an invading virus and equipping themselves to fight that virus off if it comes back again.
Specifically, when a virus infects a bacterium, the bacterium’s CRISPR reads the virus’s DNA, memorizes it, and incorporates it into its own genome. This produces RNA that Bitterman says serves as “a guide” that can recognize the virus when it comes back to infect the bacterium again, alert the Cas9 enzymes, and show them where to go. Those Cas9 enzymes then fend off infection, rendering the bacteria immune to that specific virus.
Editas says that its founders have uncovered a way to
