recreate this type of process so it can be used to “edit”—remove, fix, or otherwise—genes in human cells. Instead of there being an RNA guide that recognizes viral DNA, and sends Cas9 enzymes out to fight it, Editas would engineer its own RNA guide whose job is to recognize a specific genetic sequence—say, a corrupt or mutated gene—and transport the Cas9 enzyme to the site of the problem, where it can correct the genetic error. Bitterman says, to his knowledge, Editas is the only company so far attempting to use the CRISPR/Cas9 system to create drugs.
Editas’ founders also have another way of doing this, utilizing enzymes called transcription activator-like effector nucleases, or TALENs, but the company isn’t saying in which cases it might want to use one method over the other as of yet—just that it’s a second potential tool in the toolbox.
Though gene editing may sound similar to gene therapy, Bitterman says that the two approaches are different. Gene therapy uses engineered viruses to deliver healthy genes into a cell to replace faulty or missing ones, and is more limited.
“Gene therapy expresses a fixed copy of a broken gene,” he says. “The difference is the very precise targeting, the ability to actually impact and change or edit a gene in the genome…and to make that very specific change and nothing else.”
Editas, in theory, could use this system to create a drug that could cure any number of genetic diseases via a one-time fix, and be more flexible than gene therapy or other techniques used to cure a disease on the genetic level. But even so, the challenges, just like gene therapy, are significant. Editas has to figure out a way to safely and effectively deliver a gene-editing drug into the body, something Bitterman acknowledges is one of the big hills the company has to climb. Likely, the drugs will be delivered via injection.
“We can leverage a lot of the work that’s been done in areas like gene therapy and RNA [interference] in terms of the delivery approaches—we don’t need to reinvent the wheel,” he says. “Delivery is something we’re thinking a lot about, [and it’s] an area that our three firms and the team that we’re building knows well.”
Of course, despite its credible backing and lofty goals, Editas is in its infancy and is thus totally unproven. The best proof it has to date is evidence by its founders that the process can be engineered to work in petri dishes with mammalian cells.
Editas has five full-time employees (the goal is to get to 30 workers by the end of year two), doesn’t have an office yet, and still hasn’t decided exactly which diseases it’ll target first. The cash will be used to get the platform ready for prime time, and build out its scientific and operational team so Editas’ technology will be ready to go when the company has a better sense of what its priorities will be in clinical trials.
“It’s really time to step on the accelerator,” Bitterman says.
