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5 Diseases That Could Be Treated With CRISPR

A new sickle cell treatment opens the door for other potential gene-modifying therapies

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Danielle Del Plato

A landmark drug approval to treat a single disease may shepherd in a new era of medicine that targets multiple diseases at the genetic level.

In December, the U.S. Food and Drug Administration (FDA) approved a first-of-its-kind infusion therapy for the treatment of sickle cell disease that utilizes CRISPR, a much-talked-about gene-editing technology. CRISPR allows researchers to attack a disease by altering a patient’s genetic material. 

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Scientists often describe CRISPR as a pair of molecular “scissors,” since the tool can create a precise break in strands of DNA, the genetic code passed from parent to offspring, to alter a gene in a beneficial way.

Many diseases are caused by mutations or DNA-related problems, says Erik J. Sontheimer, chair of biomedical research and a professor at the University of Massachusetts Chan Medical School. “If we could go and actually fix the mutation in the genome [the entire set of DNA instructions found in a cell], then you would be addressing the problem at its source,” he says. A one-and-done treatment, if you will — at least that’s the idea.

“It’s really a whole new way of doing medicine,” Sontheimer says. 

Here’s a look at five conditions that could one day be treated with gene-editing therapies developed with CRISPR technology: 

1. Certain cancers

Scientists are studying whether CRISPR can help treat certain cancers, such as leukemia and multiple myeloma. In early-phase studies, researchers are modifying T cells — immune cells that fight invaders — to target and destroy cancer cells.

“You’re not actually using CRISPR on cancer cells themselves. Instead you’re making T cells with new capabilities, and the T cells are doing the therapeutic job,” Sontheimer says.

Scientists are also studying whether edited cells from a healthy donor, when delivered to someone with cancer, can successfully attack cancer cells and avoid being detected by the recipient’s own immune system. Researchers say this “off the shelf” approach is more cost- and time-efficient than custom editing each individual patient’s cells. 

2. High cholesterol

People who have genetic mutations that affect how their body regulates and removes cholesterol from the blood, a condition known as familial hypercholesterolemia, may one day benefit from CRISPR.

High cholesterol is a major risk factor for heart disease, the leading cause of death in the U.S. Heart attacks happen in 50 percent of people with untreated familial hypercholesterolemia by the age of 50, according to the Centers for Disease Control and Prevention (CDC). It’s estimated that roughly 1.3 million adults and children in the U.S. are affected by the condition.

Research presented at the 2023 American Heart Association Scientific Sessions found that a single infusion of a CRISPR-based gene-editing therapy in humans significantly reduced the “bad cholesterol,” or low-density lipoprotein cholesterol (LDL), in people who carry one gene for familial hypercholesterolemia. According to a news release from the American Heart Association, the investigational treatment works by permanently turning off a gene in the liver that plays a key role in regulating the amount of LDL cholesterol in the bloodstream.

The small, early-phase study included 10 participants, each of whom had extremely high LDL levels despite taking cholesterol-lowering medications. The participants who received the highest level of the gene-editing treatment saw reductions in their LDL levels of between 39 and 55 percent.  

“Instead of daily pills or intermittent injections over decades to lower bad cholesterol, this study reveals the potential for a new treatment option — a single-course therapy that may lead to deep LDL-C lowering for decades,” senior study author Andrew M. Bellinger, M.D., chief scientific officer of the company behind the experimental treatment, Verve Therapeutics, said in a news release.


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3. Hereditary angioedema

An early-stage study published Feb. 1 in The New England Journal of Medicine found that a single dose of a CRISPR-based therapy led to a 95 percent mean reduction of painful, unpredictable and potentially fatal swelling attacks caused by the rare genetic disease known as hereditary angioedema. The condition is estimated to affect 1 in 50,000 people.

The company behind the experimental treatment, which targets the liver and works by preventing the overproduction of an inflammatory protein that plays a key role in swelling, says it plans to launch a late-stage phase 3 trial this year.   

4. Transthyretin amyloidosis

An experimental CRISPR-based therapy for this progressive condition, characterized by the buildup of abnormally folded proteins in the body’s organs and tissues, including the nerves, heart, kidneys and eyes, is headed into late-stage clinical trials. The investigational treatment, delivered to the liver, aims to inhibit the production of the problem-causing protein by deactivating, or knocking out, the gene responsible for its production. 

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Scientists are studying ways in which CRISPR could help treat a range of infectious diseases, including HIV.  

An early-phase clinical trial is testing whether CRISPR genome-editing molecules given with a single infusion can target the virus’ genetic material and render it ineffective. Researchers working with a small number of infected adults presented initial results in October that showed no safety concerns or serious adverse events. Future studies will evaluate effectiveness.

And the list goes on. “There are a lot more diseases that are currently being explored as being amenable to CRISPR genome editing,” Sontheimer says. Scientists are even studying how CRISPR could improve the nutritional value of our food.  

“CRISPR is going to be everywhere, and in my opinion it’s going to revolutionize treatments for a lot of people — and that is speaking to more common diseases as well as rare and ultra-rare diseases,” says Laura Lambert, a consultant in the Mayo Clinic physiology and biomedical engineering department.

CRISPR research ongoing

It’s important to keep in mind that many of these potential treatments are still in early-phase trials, meaning more research is needed to confirm their safety and effectiveness. Still, Lambert predicts that “we’ll see a lot more approvals in the next five years and certainly in the next 10 years.”  

Even if these therapies do eventually receive FDA approval, costs remain a hurdle for CRISPR-based treatments. The newly approved sickle cell therapy, Casgevy, is listed at $2.2 million. “The cost right now is enormous, and it definitely raises the question of equity,” says Lambert, who notes that prices could fall once more therapies are approved. 

Delivery of CRISPR treatments also poses a challenge, scientists say. In the case of sickle cell disease and some cancers, the gene editing takes place outside of the patient’s body, and then the lab-edited cells are infused back into the patient.

Delivering a treatment so that the editing takes place inside the body is trickier. Researchers have had success in studies delivering CRISPR-based treatments to the liver and the eye. When it comes to finding effective ways to get the gene-editing tool to other organs, “a lot of ongoing research is trying to make progress there,” Sontheimer says.

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