Intellia Therapeutics is featured in Cathie Wood’s portfolio and has a high Wall Street price target.

The company’s leading candidates look somewhat promising, but one of them just hit a major setback.

Intellia Therapeutics’ prospects look very risky right now.

10 stocks we like better than Intellia Therapeutics ›

Intellia Therapeutics (NASDAQ: NTLA), a small-cap biotech company, has some notable bulls. One of them is Cathie Wood, the CEO of Ark Invest. The innovation-focused investment firm has long held shares in Intellia, likely because of its work on potentially disruptive gene-editing technology.

Intellia Therapeutics also has fans on Wall Street. The company’s current average price target of $32.3 (according to Yahoo! Finance) implies a significant 131% upside from its current levels. And that’s despite a recent setback that sent Intellia Therapeutics’ shares down significantly.

Should investors buy the stock at current levels? Let’s find out.

Intellia Therapeutics focuses on developing therapies for rare diseases. The company has two products in clinical trials, both CRISPR-based medicines. One of them is Lonvo-z, being developed to treat hereditary angioedema (HAE), a genetic disease that causes unpredictable episodes of swelling.

Lonvo-z could be a onetime cure for this condition. And as an in vivo gene-editing therapy, it avoids the complex cell collection and editing process that often makes ex vivo therapies so challenging to administer.

Lonvo-z performed well in early-stage studies and is now in a phase 3 clinical trial, with data from this study and a potential regulatory submission expected next year.

Image source: Getty Images.

Then there is nex-z, a medicine Intellia Therapeutics is developing in collaboration with Regeneron Pharmaceuticals. The partners hope that nex-z can treat transthyretin (ATTR) amyloidosis, a disease caused by a dangerous buildup of the transthyretin protein in the body (in the heart or around certain organs). This can lead to a range of health problems, such as cardiomyopathy, or when the heart can’t properly pump blood, and polyneuropathy, or peripheral nerve damage.

Nex-z is undergoing two phase 3 studies: one in patients with ATTR amyloidosis and cardiomyopathy, and the other targeting patients with polyneuropathy. However, the company recently announced it had to pause these clinical trials because one patient suffered from significant liver damage. The patient has not died and is being treated, but this raises serious questions about whether nex-z is safe and will ever earn regulatory approval.

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