The fields of gene editing and gene therapy have been growing since the first draft of the human genome map was finished in 2003. In 2022, gaps in that map were completed. But even before then, the roadmap gave researchers the opportunity to develop gene therapies to correct genetic abnormalities.
Gene editing, which seeks to directly alter a patient’s genome itself, is the next stage. If successful, it may provide a viable treatment for many previously untreatable genetic disorders. That is why getting in on the ground floor of these top three biotech stocks could be so beneficial for long-term investors.
Several of these companies that are at the forefront of gene editing have partnerships with established, large-cap biotech companies. This helps fund the projects, and the payoff appears to be closer than ever. The U.S. Food & Drug Administration (FDA) recently approved gene editing as a viable treatment option so this market is expected to grow quickly, potentially to $21.3 billion by 2030.
Here are three gene-editing stocks that are leading the way in this sector.
CRISPR Therapeutics (CRSP)
CRISPR Therapeutics (NASDAQ:CRSP) is synonymous with gene editing. One of the company’s founders, Dr. Emmanuelle Charpentier, is the co-inventor of the CRISPR-Cas9 gene-editing tool. However, it’s been over a decade and the company still does not have a treatment on the market.
That is likely to change soon. In partnership with Vertex Pharmaceuticals (NASDAQ:VRTX), the company is getting close to having FDA approval for its exa-cel candidate as a one-time treatment for sickle cell disease and beta thalassemia.
The company recently received Prescription Drug User Fee Act (PDUFA) action dates from the FDA. The first action date will be in December 2023. This will cover exa-cel’s indication for sickle cell disease. In March 2024, the next action date will cover the indication for beta thalassemia.
Approval for exa-cel is just the tip of the iceberg, as the company has a long pipeline of potential treatments including those for type 1 diabetes, immuno-oncology and cardiovascular disease.
Perhaps in expectation of this positive news, CRSP stock is up over 30% year-to-date (YTD). The average consensus price target of 25 analysts is $86.68 which is a 66.12% increase from the current CRSP stock price.
Editas Medicine (EDIT)
Editas Medicine (NASDAQ:EDIT) is one of many gene-editing companies that are using CRISPR technology to develop their own therapies. Its lead candidate, EDIT-301, is in late-stage clinical trials. Like CRISPR’s exa-cel, EDIT-301 is a treatment for sickle cell disease and beta thalassemia.
The company appears it may have enough cash on hand to get EDIT-301 approved. But with the company burning significant cash with only a small amount of revenue coming in the door, investors should make sure to stay informed when purchasing this biotech stock.
That being said, like CRSP stock, analysts are still bullish on Editas. The average consensus price target for EDIT stock shows the potential for a 66.79% gain. This could be a race between Editas and CRSPR for approval. And for those that are wondering, courts have ruled in favor of Editas in terms of its intellectual property involving its use of CRISPR technology.
Intellia Therapeutics (NTLA)
Intellia Therapeutics (NASDAQ:NTLA) is another company using CRSPR technology, but its lead candidate, NTLA-2001 targets transthyretin (ATTR) amyloidosis. There may be a $9.35 billion opportunity for therapeutic treatments by 2030. The company also has NTLA-2002 in late-stage clinical trials as a potential gene-editing treatment for hereditary angioedema.
Intellia is developing NTLA-2001 in partnership with Regeneron Pharmaceuticals (NASDAQ:REGN), another biotech stock to keep an eye on. NTLA-2001 is the first CRISPR therapy designed to be administered intravenously in humans. In October 2023, Intellia received FDA approval to advance NTLA-2001 into Phase 3 trials that are expected to begin by the end of the year.
The consensus price target for NTLA stock is $82.96, a 230.52% increase from the stock’s current price.
On the date of publication, Chris Markoch did not hold (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.