Cathie Wood's $362 Million CRISPR Therapeutics Bet Isn't Just Bold -- It's Backed by a Catalyst Many Investors Are Underrating

If you're keeping an eye on gene-editing specialist CRISPR Therapeutics (NASDAQ: CRSP), you probably already know the up-and-coming biopharmaceutical outfit is a favorite of Ark Investment Management's CEO and chief stock picker, Cathie Wood. As of the latest look, the company's holding nearly $270 million worth of this name in its flagship Ark Innovation ETF (NYSEMKT: ARKK) and another $92 million worth in the smaller Ark Genomic Revolution ETF (NYSEMKT: ARKG).

What is surprising is why Wood is holding it. Although patient-specific genomic repair remains a key part of this stock's bullish thesis, CRISPR Therapeutics' developmental work on another front has gone largely unnoticed and may not be reflected in the stock's price.

CRISPR Therapeutics' other R&D work

CRISPR Therapeutics' founders essentially found a way of repairing damaged DNA with a corrected genetic sequence. It was the first company to ever win the FDA's approval for a gene-editing drug, in fact. That's Casgevy -- for the treatment of sickle cell disease -- which was approved in late 2023. Now the same patient-specific approach is being tested as a therapy for handful of other genetic diseases.

The underlying science, however, isn't limited to a customized therapy for each patient. Gene editing can be used to create off-the-shelf treatments for all patients with a particular disease.

Image source: Getty Images.

That's something CRISPR Therapeutics has been quietly working on for a while now, developing a pair of noteworthy chimeric antigen receptor (CAR) T-cell therapies. Its CTX110 is currently in preclinical testing stages, although its zugocabtagene geleucel -- formerly CTX112 -- is now in phase 1 clinical trials, where it's showing strong promise as a lymphoma treatment as well as a therapy for autoimmune diseases like lupus, systemic sclerosis, and inflammatory myositis.

And again, unlike Casgevy and some of the other drugs in its pipeline, CRISPR's CAR T-cell therapy program doesn't take months and requires a sample of the patient's own cells. Any healthy donor can provide the T-cells needed to target the CD19 protein, often found on cancerous cells and cells associated with autoimmune conditions. This cuts down on its costs, not just for CRISPR, but also for the patient and/or the patient's insurer.

CAR-T therapies are one of the pharmaceutical industry's next big frontiers. Global Market Insights suggests the CAR T-cell therapy market is poised to grow at an average annual pace of more than 30% through 2034, when it could be worth more than $60 billion per year.

Risk worth managing

CRISPR Therapeutics isn't the only biopharma name aiming at this future business. Major players like Novartis, Bristol Myers Squibb, and Gilead Sciences already have CAR T-cell therapy drugs on the market and are developing more. CRISPR's CAR T-cell therapy trials are also in their earliest stages. They won't be ready for an approval request until the latter portion of Global Market Insights' projection period.

Still, Cathie Wood seems to understand that the market can and will reward progress along the way. If you're interested, just do what she does and make your stake part of a well-diversified portfolio.

James Brumley has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Bristol Myers Squibb and Gilead Sciences. The Motley Fool recommends CRISPR Therapeutics. The Motley Fool has a disclosure policy.