What happened

Vertex Pharmaceuticals (VRTX 0.20%), a biopharmaceutical company that specializes in gene-editing therapies, saw its shares rise 2.8% in September, according to data provided by S&P Global Market Intelligence. Normally, that wouldn't be worth that much of a note, but this was the same month that the Dow Jones Industrial Average fell 9.2%, roughly 3,000 points. Vertex easily went against the tide, though.

It closed August at $281.36, then rose to a high of $296.14 on Sept. 12, before closing the month at $289.54. It is really part of an upward trend for the stock, which is up more than 36% in 2022. It has a 52-week low of $176.36 and a 52-week high of $305.95.

So what

Vertex, known for its franchise of therapies to treat cystic fibrosis (CF), a rare genetic disorder that affects the lungs, pancreas, liver, kidneys, and intestines, showed that it is about to expand its portfolio of marketed drugs. The company is working with CRISPR Therapeutics in developing exa-cel, a one-time curative gene therapy for two genetic blood disorders that require transfusions: beta-thalassemia and sickle cell disease.

The company reported that it is on track to submit exa-cel's approval for rolling review to the Food and Drug Administration starting in November, with plans of completing the new drug application submission by the first quarter of 2023. It also said that submissions in Europe are on track for as early as the fourth quarter of this year.

This means that beginning next year, Vertex has a potentially very lucrative curative therapy that it will be able to sell.

On top of that, Vertex is already in a solid position financially, thanks to its main CF therapy, Trikafta. In the second quarter, the company reported $2.2 billion in revenue, up 12% year over year. Trikafta was responsible for $1.9 billion of that, and its own revenue was up 50% over the same period last year. The company reported earnings per share of $3.13, compared to $0.26 in the same quarter a year ago.

Now what

At this point, much of the excitement around Vertex may already be baked into the biotech stock's price. Investors will wait to see how the review process goes for exa-cel and a delay in that process could drop the stock a bit. In the long run, however, it certainly seems that Vertex has turned a corner and has shown that it and CRISPR Therapeutics are the first companies that are capable of turning a CRISPR-editing therapy into a marketed therapy.