The gene editing technology CRISPR reached an important milestone last weekend and completed its first systemic drug delivery to the human body.
CRISPR, or clustered, regularly spaced short palindromic repeats, cuts genomes and cuts DNA effectively to treat genetic diseases.
The latest breakthrough, the result of a study between biotech company Regeneron and Boston startup Intellia Therapeutics, treats a rare disease after it has been given as an IV infusion. Previously, other uses of CRISPR technology have been limited to ex vivo therapy, or where cells are removed from the body for genetic manipulation in a laboratory and then returned to the body.
Jennifer Doudna, who was awarded the Nobel Prize in Chemistry in 2020 for her work on CRISPR gene editing and is the co-founder of Intellia, recently told CNBC about the development of the technology from the publication of her early work to clinical studies showing its effectiveness at treating disease in less than 10 years means “one of the fastest rollouts in my opinion of technology from basic, initial science to actual application.”
“This is mainly because the technology comes at a time when there is a huge demand for genome editing and a lot of knowledge about genomes,” said Doudna at the recent CNBC Global Evolve Summit in mid-June.
As for next, Doudna highlighted several challenges and opportunities that CRISPR has on the horizon.
Deploying CRISPR remains a major challenge
As technology continues to advance, the task of getting the processed molecules in the body to the cells in the areas where they are needed remains a challenge.
“This is a particular issue in clinical medicine where the ability to manipulate brain cells, heart cells or muscle cells has incredible potential, but right now we don’t really have the tools to introduce the editors to those cells,” said Doudna. “We have the editors; we just don’t know how to get them where they need to go.”
Sickle cell anemia was an early focus
Much of the success of CRISPR’s previous applications has been in ex vivo therapy, in which extracted cells are manipulated in a laboratory and then returned to a patient.
Sickle cell anemia, which is genetically inherited and affects approximately 100,000 Americans, according to the CDC, was a particularly good target for the technology because blood stem cells “can be harvested, processed, and then returned to patients,” Doudna said.
Genetic eye diseases were also a focus for CRISPR applications, as Doudna said, “It is certainly easier to get into the eye than other parts of the body.”
The delivery of the processed cells to the liver has also proven to be easier so far. “A liver is an organ that naturally accepts molecules in the body,” she said.
Any advance in eradicating the 100+ liver diseases could have a huge impact on the lives of Americans. According to the American Liver Foundation, at least 30 million people, or one in ten Americans, have liver disease.
Next, let’s focus on the brain, heart, muscles
The next step for innovation around CRISPR will be to move these cells to other parts of the body like the brain, heart and muscles, Doudna said.
“There are already some technologies that make some of this possible, for example with different types of viruses or virus-like particles, and I look forward to the innovations that will come in this regard over the next few years,” she said.
Treatment costs are a problem
But as technology improves and scientists become able to fight disease throughout the body, Doudna said that CRISPR technology needs to be cheaper in order for it to have “widespread effects.”
Treating sickle cell anemia with CRISPR therapy costs about $ 2 million per patient, according to Doudna.
“That is clearly not a price point that makes this available to most of the people who can benefit from it,” she said.
While addressing delivery challenges can also help reduce costs, the medical community needs to figure out how “to scale molecule production so that we can cut costs,” said Doudna.
Applying CRISPR to Agriculture
The advancement of CRISPR technology may have an impact on other industries as well, with agriculture being one of the first to benefit.
Rather than addressing genetic problems through breeding, which can take months to years, or through current methods of genetically modifying crops that have seen a boom over the past few decades but that incorporate biological material from other species, CRISPR technology can address the Genes from plants “without touch” manipulate everything else, “said Doudna.
“This opens the door to a lot of things that can be done now to both address the challenges of climate change, manage drought, and introduce properties into the plants that will protect them from pests,” she said.
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