The Next Moderna? This Clinical-Stage Biotech Has Breakout Potential.
Another such clinical-stage contender generating growing excitement is Intellia Therapeutics (NTLA), a CRISPR/Cas9-based gene-editing company. Intellia aims to provide one-time, curative treatments for serious genetic diseases. Intellia's stock has fallen 1.9% year-to-date, while the broader market, as tracked by the S&P 500 Index ($SPX), has risen 6%.
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Let's dig deeper to see if this could be the next transformative biotech story that reshapes medicine — and markets — in the same way Moderna did.
Intellia employs a dual-platform strategy, focusing on both in vivo (inside the patient's body) and ex vivo (outside the patient's body) approaches to treating conditions such as transthyretin amyloidosis (ATTR) and hereditary angioedema (HAE). Intellia's lead in vivo therapies include NTLA-2001 (Nexiguran ziclumeran or nex-z), a treatment for ATTR amyloidosis that is in the Phase 3 MAGNITUDE trial. Nex Z was also granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA), indicating its potential for accelerated development.
NTLA-2002, a treatment for hereditary angioedema (HAE), is now in late-stage clinical trials. In the first quarter of 2025, Intellia administered the first dose in the HAELO trial. This event marks a critical inflection point, bringing Intellia closer to regulatory filings and possible approval. However, the company recently halted its NTLA-3001 lung disease candidate, as well as several other research programs. It also reduced its workforce by about 27%, causing a short-term disruption. The goal of this strategic reorganization is to increase focus on late-stage assets while conserving capital.
While Intellia does not yet generate product revenue, it did generate $16.6 million in collaboration revenue during the first quarter. As of Q1 end, the company held $707.1 million in cash and executed a 27% workforce reduction, ensuring a runway until the first half of 2027.
Intellia's first-mover advantage in in vivo gene editing could propel it to great success if its candidates are approved. The company has also collaborated with heavyweights like Regeneron (REGN), gaining expertise, resources, and a broader clinical reach.
However, as a clinical-stage biotech company, the company faces the risk of clinical trials failing or delays in regulatory approvals because gene editing is a high-stakes game. Furthermore, increased cash burn may necessitate dilution. Long-term growth investors who believe in CRISPR's transformative potential may view NTLA stock as an enticing but high-risk investment opportunity.
Overall, Wall Street has assigned Intellia stock a 'Moderate Buy' rating. Of the 27 analysts covering NTLA stock, 19 have a 'Strong Buy' recommendation, one suggests a 'Moderate Buy,' six say it is a 'Hold,' and one suggests a 'Strong Sell.' The average price target of $38.04 implies the stock has upside potential of 245% over current levels. Plus, the Street-high price estimate of $106 suggests the stock can rally over 860% over the next 12 months.
While the upside appears out of reach, Moderna stock is a prime example of how a single successful product can propel a biotech stock toward the skies. Intellia, like Moderna, focuses on diseases with high clinical need and, if successful, has the potential to transform medicine.
On the date of publication, Sushree Mohanty did not have (either directly or indirectly) positions in any of the securities mentioned in this article. All information and data in this article is solely for informational purposes. This article was originally published on Barchart.com
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