CRISPR -- which stands for "clustered regularly interspaced short palindromic repeats" -- has been a hot area of research and investment since scientists discovered that this naturally occurring gene-editing function of bacteria could conceivably be used to treat genetic diseases. Several companies are using gene-editing in their attempts to cure illnesses caused by errors on a single gene such as sickle cell disease, hemophilia, and cystic fibrosis. One of them, CRISPR Therapeutics (NASDAQ:CRSP), has produced results that could not only make it a winner in single-gene disorders, but position it to tackle much more complex -- and profitable -- diseases in the years ahead.

First things first

CTX001 is the company's candidate treatment for sickle cell disease and beta-thalassemia -- two disorders that affect the oxygen-carrying cells in our blood. Medical professionals harvest a patient's own cells from bone marrow, use CTX001 to edit the gene affecting red blood cell production, and infuse the cells back into the body.

In 2015, CRISPR Therapeutics formed a partnership with Vertex Pharmaceuticals (NASDAQ:VRTX) to develop a number of treatments using this technology, accepting cash, equity, and future royalties. In exchange, Vertex got the rights to market the treatments developed. 

A doctor using small scissors to cut out a portion of the double helix DNA strand.

Image source: Getty Images

So far, the collaboration has produced outstanding results. At the recent American Society of Hematology conference, the companies reported on 10 patients who had been treated with CTX001. All patients had been free of the negative events associated with the diseases -- the need for transfusions in the case of beta-thalassemia, and pain crises for sickle cell disease. Data on another 10 patients will be reported out in 2021. While the science is transformational, the market for this particular treatment isn't huge. About 300,000 people are born each year with sickle cell disease, and 60,000 with beta-thalassemia.

A promising pipeline

Single-gene disorders aren't the only programs in CRISPR Therapeutics' pipeline. It's currently enrolling participants in studies testing CAR-T therapies, which use gene-editing to give immune cells the ability to recognize and attack specific types of cancer cells.

One program, CTX110, has already shown dose-dependent reduction in tumor sizes, and patients' responses to the treatment have lasted beyond three months. Despite the fact that the CRISPR-related hype over the last few years has outraced its success, these programs are proving the technology has real healing power.

The company has also partnered with private company ViaCyte to develop a treatment for Type 1 diabetes. The companies hope gene-editing can protect ViaCyte's replacement pancreatic stem cells from the body's immune system. According to the companies, results from a phase 1/2 clinical trial of that treatment are expected to be released in 2021.

Medicine in the year 2030

In the near term, the company has already laid out plans for research collaborations with Vertex on Duchenne muscular dystrophy, cystic fibrosis, and they have plans to address other diseases they are not yet making public.

And the most profound applications in this arena may not involve editing genes, but rather in turning gene expression on or off. The idea of using CRISPR to temporarily turn off the cell receptors that a particular virus binds to, for example, or to silence the genes responsible for high cholesterol production, have been on researchers' minds since the technology was discovered. 

Researchers have begun to write CRISPR "programs" where actions are taken on multiple genes at once. This has already been done to turn stem cells into neurons -- the cells that make up the central nervous system. Imagine a treatment that could create new healthy cells to replace those that function improperly in someone with Alzheimer's or ALS.

One of the hurdles so far has been getting the edited genes into the patients' cells, but scientists have been working on that too. Evidence of their successes on that front can be found in two of the highest-profile COVID-19 vaccine candidates. The vaccines use synthetic messenger RNA (mRNA) to deliver instructions to cells, prepping them to fight the disease should it come around. The newly advanced mRNA technology could combine with CRISPR to transform how we fight diseases.

A decade from now, drug companies may be using their expertise in the biology of a disease to license the genetic technology to edit or activate the genes necessary to produce the desired cellular function. They would then deliver those treatments via synthetic mRNA.

Similar to cellphone makers that license Google's Android operating system to run their hardware, drug developers may need a standard platform for performing tasks at the genetic level. So far, CRISPR Therapeutics is making a strong case to be the differentiated platform that launches a new era in medicine. The stock of any biotech company is likely to be volatile over shorter periods, but CRISPR Therapeutics could handsomely reward investors who have long time horizons.



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