Small biopharma story stocks just aren't paying off like they used to. Maybe there are just too many of them, with each one working on a medical breakthrough that's statistically unlikely to even come close to an approval.
Every now and then, though, one of these companies defies the odds and gets a new drug on the market. CRISPR Therapeutics (CRSP +1.58%) is one of those companies, and what a game changer it introduced!
A whole new kind of drug
The pharmaceutical industry has been working on DNA repair for years, albeit with little marketable success. That all changed in late 2023, however, when CRISPR Therapeutics' and developmental partner Vertex's drug Casgevy -- for the treatment of sickle cell disease -- became the FDA's first-ever approved CRISPR-based gene-editing therapy.
Image source: Getty Images.
In and of itself it doesn't mean much; sickle cell disease isn't exactly a major market.
The fact that the U.S. Food and Drug Administration approved any CRISPR (clustered regularly interspaced short palindromic repeats) drug is significant, however. It paves the way for similar gene-editing therapies aimed at more widespread diseases.
To this end, CRISPR Therapeutics is using Casgevy's underlying science in six other drug trials, two of which are oncology drugs, and two others of which are meant to treat heart disease. And they could be huge. An outlook from Straits Research suggests the worldwide CRISPR-based gene-editing drug market alone could grow at an average pace of nearly 25% per year all the way through 2033.
Leading the way
CRISPR Therapeutics isn't the only name in the gene-editing space. Privately owned Bluebird Bio's Zynteglo is a genetic-repair therapy also now approved to treat sickle cell disease as well as beta thalassemia. Beam Therapeutics and Intellia Therapeutics are two more promising names in this particular race, which is starting to get a bit crowded now that major developers see this science's potential opportunity.
CRISPR Therapeutics was the first to market, though, and is arguably still leading the developmental race in CRISPR technology. Analysts expect the company's revenue this year to quadruple next year, driven by growing demand for Casgevy.
