If you have been following gene-editing specialist CRISPR Therapeutics (CRSP), you likely already know that this emerging biopharmaceutical company is a favored pick of Cathie Wood, CEO and chief stock picker of Ark Investment Management. According to the latest data, the firm holds nearly $270 million worth of CRISPR Therapeutics stock in its flagship Ark Innovation ETF (ARKK) and an additional $92 million in the smaller Ark Genomic Revolution ETF (ARKG).
What is truly thought-provoking is the reason Wood holds this stock. Although patient-specific genomic repair remains a key part of the bullish thesis for the stock, CRISPR Therapeutics’ research and development work in another frontier area has largely flown under the radar and may not yet be reflected in the share price.
CRISPR Therapeutics’ pipeline includes an allogeneic chimeric antigen receptor T-cell (CAR-T) therapy—CTX112—currently in clinical testing. Allogeneic (referring to genetically distinct tissues or cells) CAR-T therapies are often called “off-the-shelf” therapies because they do not require gene editing of a patient’s own cells, instead using cells from healthy donors. CRISPR Therapeutics is evaluating several in vivo gene-editing therapies, two of which have entered clinical studies. The company is also conducting clinical trials on an experimental therapy for the treatment of type 1 diabetes.
Gene editing can be used to create “off-the-shelf” treatment regimens for all patients with a given disease. This is exactly what CRISPR Therapeutics has been quietly advancing for some time, developing two noteworthy CAR-T cell therapies. Its CTX110 is currently in preclinical testing, while zugocabtagene geleucel (formerly CTX112) has now entered Phase 1 clinical trials, showing promising potential as a therapy for lymphoma as well as for autoimmune diseases such as lupus, systemic sclerosis, and inflammatory myositis.
Unlike Casgevy and some other drugs in its pipeline, CRISPR’s CAR-T cell therapy programs do not require months-long timelines or the collection of a patient’s own cell samples. Any healthy donor can provide the T cells needed to target the CD19 protein, which is typically found on cancer cells and cells associated with autoimmune diseases. This lowers costs, not only for CRISPR but also for patients and/or their insurers.
CAR-T therapy represents the next major frontier in the pharmaceutical industry. Global Market Insights estimates that the CAR-T cell therapy market will grow at an average annual rate of more than 30% through 2034, by which time the annual market size could exceed $60 billion.
CRISPR Therapeutics is not the only biopharmaceutical company targeting this future business. Major pharmaceutical firms such as Novartis, Bristol-Myers Squibb, and Gilead Sciences already have CAR-T therapy drugs on the market and are developing additional products. CRISPR’s CAR-T therapy trials are also at the earliest stages, and these therapies will not be ready for submission of marketing applications until the latter half of Global Market Insights’ forecast period.
Nevertheless, Cathie Wood seems to understand that the market can and will reward progress as it advances. If you are interested, consider following her lead by holding your position as part of a fully diversified portfolio.
