Gene-editing technology CRISPR reached a major milestone this past weekend, completing its first systemic delivery as a medicine to a human body.
CRISPR, or clustered regularly interspaced short palindromic repeats, effectively cuts genomes and slices DNA to treat genetic diseases.
The latest breakthrough, the result of a trial between biotech company Regeneron and Boston-based startup Intellia Therapeutics, treated a rare disease after being given as an IV infusion. Previously, other applications of the CRISPR technology had been limited to ex vivo therapy, or where cells are removed from the body for genetic manipulation in a laboratory and then reintroduced to the body.
Jennifer Doudna, who was awarded the 2020 Nobel Prize in chemistry for her work on CRISPR gene editing and is the co-founder of Intellia, recently told CNBC the evolution of the technology from the publication of her early work to clinical trials showing it to be effective in treating diseases in less than 10 years represents, “One of the fastest rollouts I think of technology from the fundamental, initial science to an actual application.”
“It’s largely because the technology comes at a moment when there’s enormous demand for genome editing, as well as a lot of knowledge about genomes,” Doudna said at the recent CNBC Global Evolve Summit in mid-June.
As for what’s next, Doudna highlighted several challenges and opportunities on the horizon for CRISPR.
While the technology has continued to advance, the task of getting the edited molecules to travel in the body to the cells in the areas where they are needed remains a challenge.
“This is especially an issue in clinical medicine where being able to edit brain cells, heart cells or muscle cells has incredible potential but right now we don’t really have the tools to introduce the editors into those cells,” Doudna said. “We have the editors; we just don’t know how to get them where they need to go.”
Much of the success of the applications of CRISPR thus far has been with ex vivo therapy, where extracted cells are manipulated in a laboratory and then reintroduced into a patient.
Sickle cell anemia, which is passed down genetically and affects approximately 100,000 Americans, according to the CDC, has been a particularly good target for the technology as blood stem cells can be “harvested, edited and then reintroduced to patients,” Doudna said.
Genetic diseases of the eye have also been a focus for CRISPR applications as Doudna said “it’s easier certainly to deliver to the eye than to other parts of the body.”
Delivering the edited cells to the liver has also proven to be easier thus far. “A liver is an organ that naturally takes up molecules in the body,” she said.
Any progress in eradicating the more than 100 liver diseases could have a major impact on the lives of Americans. At least 30 million people, or one in 10 Americans, has some sort of liver disease, according to the American Liver Foundation.
The next step for innovation around CRISPR will be getting those cells to other parts of the body, such as the brain, the heart and muscles, Doudna said.
“There are some technologies already that enable some of this, for example using various kinds of viruses or virus-like particles, and I’m excited about the innovation that will come in the next few years in this regard,” she said.
But as the technology improves and scientists gain the ability to target diseases all across the body, Doudna said that for CRISPR technology to be “widely impactful,” it will need to be cheaper.
Treating sickle cell disease with CRISPR therapy, Doudna said, costs about $2 million a patient.
“That is clearly not a price point that will make this available to most people that can benefit from it,” she said.
While addressing delivery challenges may also help lower costs, Doudna said that the medical field needs to figure out how to “scale the molecule production so that we reduce costs.”
The advancement of CRISPR technology can also have an impact on other industries, with agriculture being one of the first to benefit.
Rather than trying to address genetic issues through breeding which can take months to years, or current methods for genetically modifying crops that have boomed in recent decades but involve inserting biological material from other species, the CRISPR technology can manipulate the genes of plants “without touching anything else,” Doudna said.
“This is opening the door to lots of things now that can be done to both address challenges of climate change, dealing with drought conditions, introducing traits in the plants that give them protection against pests,” she said.