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The 5 most promising uses for CRISPR gene-editing technology so far
From cancer to HIV, these are some of the areas where CRISPR gene-editing drugs show the most promise, say scientists.
The concept of CRISPR gene-editing is still quite young. Although the discovery of CRISPRs themselves—repeating DNA sequences present in single-celled organisms, such as bacteria—can be traced back to the late 1980s, their use in more complex multi-celled organisms for the purposes of targeted gene-editing and the slicing-and-dicing of genetic code to combat diseases and transform life forms only goes back to 2012 and the work of such scientists as George Church, Fengh Zhang, and Nobel Prize winners Jennifer Doudna and Emmanuelle Charpentier.
In that short decade, the application of CRISPR to treat diseases running the gamut from forms of inherited blindness to blood disorders to cancers and beyond has been a hotspot of biopharmaceutical innovation. At the same time, underlying CRISPR technologies have evolved to perform more sophisticated gene-editing that functions less like molecular scissors cutting out aberrant genetic code and more like a keyboard, with a “search-and-replace” function (dubbed “prime editing” in CRISPR 3.0) that can swap out one molecular letter for another.
The technological evolution could open up a host of new medical possibilities for CRISPR going forward. For now, here’s where we’ve seen the most progress, and the biggest potential, for the use of this kind of gene-editing in the medical world.
BLOOD DISORDERS
One of the two biggest CRISPR-related developments of the year—and one of the more eye-catching drug development breakthroughs in recent memory—involves its use in the genetic blood disorders beta thalassemia and sickle cell disease (SCD). These are not extremely common conditions in the U.S. since, as genetic conditions, they afflict certain populations, and therefore, some geographies more than others. Worldwide, however, the conditions are far more prevalent, preventing the proper transport of oxygen throughout the body because of missing genes and malformed red blood cells that lead to major problems in multiple organs and need for regular blood infusions.
To that end, rare-disease drug maker Vertex Pharmaceuticals and CRISPR Therapeutics, one of the original biotechs to focus on the technology, announced promising data earlier this year that set the companies up to become the first to have an FDA-approved CRISPR-based therapy, called exa-cel on the market as soon as mid-to-late 2023. By the end of September 2022, the companies had submitted the drug for FDA approval.
CANCER
Another critical 2022 development in the CRISPR space targets one of medicine’s everlasting holy grails: better ways to fight cancer.
A small clinical study published in the journal Nature this past year found that CRISPR gene-editing can be leveraged to alter the immune system, guiding T cells to attack specific tumor types by helping them recognize and home in on certain biological markers on those tumors.
Effectively, this could be used to create a precisely targeted arsenal against solid tumors in various cancers, such as breast and colon, according to the study authors, and kill them off.
“It is probably the most complicated therapy ever attempted in the clinic,” study coauthor, physician, and cancer researcher Antoni Ribas of the University of California, Los Angeles told Nature in an interview on the study. “We’re trying to make an army out of a patient’s own T cells.”
HIV
Excision BioTherapeutics launched a landmark trial in humans this year in search of a functional cure for HIV. This one, however, has a twist: Unlike other deployments of CRISPR that edit the building blocks of human cells, this tactic targets the HIV virus itself, snipping out the regions that would allow the virus to replicate in the first place.
In this instance, the goal is to eliminate the ability of HIV to persist at all in the human body. There have already been several big achievements in HIV treatment that have made it a relatively manageable chronic condition with the use of antiretroviral drugs; Excision’s goal is to take away the need for even that level of maintenance.
RARE DISORDERS AND BEYOND
“The targets we’re finding with CRISPR . . . are going to guide the drugs coming out in the 2020s,” said Jon Moore, chief scientific officer at biotechnology company Horizon Discovery, all the way back in 2016. That certainly seems to be the case, given current CRISPR-related experimental drug pipelines. From ALS (also known as Lou Gehrig’s disease) to rare conditions, such as transthyretin amyloidosis (ATTR) which can cause heart failure, and more common afflictions, such as urinary tract infections caused by bacteria (which Locus Biosciences is targeting with a CRISPR-based therapy), the life-sciences industry is in the early stages of finding the science fiction—reminiscent technology’s best use cases. Just like any new technology, not all of them are bound for success—but the progress of companies like Vertex, CRISPR, and Bluebird bio in the past year alone show that the industry is putting significant bets on the future of CRISPR.
