The findings for the first-ever human trial that uses CRISPR gene-editing technology to alter the immune cells of cancer patients have been announced.
The trial, which is hailed as the first of its kind to ever publish its results, centered on three patients suffering from advanced cancer who are all in their 60s. The goal is to determine whether or not their bodies could tolerate the genetically edited immune cells.
The patients received doses of CRISPR-modified variants gathered from their own T cells, which were specifically edited to transform into more efficient cancer-killing cells.
The results showed that there were no issues reintroducing the edited cells back into the bodies of the patients. More impressively, the modified cells managed to survive longer than the anticipated period.
In fact, these cells were detected in the patients’ bodies nine months following the novel treatment.
Doctors also noted that the patients’ symptoms stabilized throughout the treatment period. One of them even saw a reduction in tumor size.
While the treatment was only a one-time injection and was not carried on for a longer time, the fact that no major complication happened during the trial has health experts hailing it a success. Hence, more trials of this nature can be expected in the near future.
As expected, this trial boosted gene-editing stocks -- and CRISPR Therapeutics (CRSP) is one of the beneficiaries of this positive news.
This development is anticipated to further fuel investor interest in CRISPR Therapeutics especially after it released an impressive fourth-quarter financial report that beat revenue expectations.
The company’s profits grew to $77 million, indicating a substantial jump from the measly $100,000 it reported in the fourth quarter of 2018. As for its cash and cash equivalents, the amount increased by 106.7% from $456.6 million last year to $943.8 million.
Meanwhile, its total annual income increased from $3.1 million to a whopping $289.6 million.
A quick look at the changes done by the company revealed that the surge can be mostly attributed to CRISPR Therapeutics’ collaboration with Vertex Pharmaceuticals (VRTX) and not product sales.
Nonetheless, the improvement in the gene-editing company’s performance is still impressive considering that analysts only estimated their earnings to reach $45.2 million in the said quarter.
While these numbers are already turning heads, CRISPR Therapeutics is expected to dominate more headlines in 2020.
So far, the company has four major treatments in development.
One is called CTX001, which is for genetic blood disorders specifically sickle cell disease and transfusion-dependent beta thalassemia. Results involving this treatment should be out sometime this year.
The other three, CTX110, CTX120, and CTX130, are cancer treatments commonly known as CAR-T therapies.
CRISPR Therapeutics is an obvious leader in the race to commercialize CRISPR/Cas9 gene-editing services and products.
The lowdown is that its treatments under development, which involve groundbreaking innovations focused on rare diseases, have the potential to turn in hundreds of billions in sales. More impressively, CRISPR Therapeutics is poised to achieve this in record time --- way ahead of its competitors.
So, what’s the catch?
Well, CRISPR Therapeutics’ whole platform could end up amounting to nothing more than a fascinating science experiment. If that happens, then this stock would be worthless.
However, Vertex Pharmaceuticals has a stellar track record of picking winners. Its decision to splurge on CRISPR Therapeutics and back the latter’s research speaks volumes of the mid-cap biotechnology company’s potential to turn into a frontrunner in this novel world of gene editing.
Needless to say, CRISPR Therapeutics’ current valuation arguably indicates a once-in-a-lifetime buying opportunity. However, this high-risk investment would only appeal to aggressive investors.
