Gene editing is a game-changer. With it, we can remove cells from your body, edit them, and then reintroduce them back into the body. All to treat cancers, blood issues, blindness, cystic fibrosis, and muscular dystrophy. All of which is creating substantial opportunities for some of the leading gene editing stocks.
What’s more exciting is companies like Profluent Bio “leveraged artificial intelligence to design an open-source gene editor called OpenCRISPR-1, demonstrating the technology can be used to create molecules with the power to edit human DNA,” as noted by MobiHealthNews.com.Even better, analysts at MarketsandMarkets say the market could be worth nearly $10.6 billion by 2028 from $5.3 billion in 2023.
Plus, At the moment, there are about 7,000 diseases caused by genetic disorders. All of which occur when a mutation affects your genes. Or, when you have the wrong amount of genetic material, as noted by the Cleveland Clinic. Many could potentially be fixed is the hope.
With that, investors may want to jump into some of the top gene-editing stocks, including:
Top Gene Editing Stocks: CRISPR Therapeutics (CRSP)
In early Dec. 2023, CRISPR Therapeutics (NASDAQ:CRSP) received Food & Drug Administration approval for its sickle-cell treatment. It also received clearance for its gene-edited therapy for transfusion-dependent beta-thalassemia (TDT).
Both of which sent the stock to a high of about $91 by Feb. 2024.
While it’s still one of the most exciting gene editing stocks, its earnings miss dragged CRSP back to $55.95. However, the weakness is a buying opportunity.
For one, according to analysts at Wells Fargo, CRSP could be a takeover target with its therapies. Two, analysts at Baird recently raised its price target on CRSP to $52 from $46. All in anticipation of new clinical data from its CAR-T pipeline.
Its CTX112, for example, is now in a Phase 1/2 trial for CD19-positive relapsed or refractory B-cell malignancies, with the company expected to post preliminary data this year. Its CTX131, being developed for solid tumors is also heading into hematologic malignancies this year.
Intellia Therapeutics (NTLA)
Beaten-down shares of Intellia Therapeutics (NASDAQ:NTLA) have been on the mend after dropping from about $33 to $19.37. Now at $24, I’d like to see it climb back to $33 initially.
Helping, the company just “presented new data demonstrating for the first time the potential for redosing with an investigational, in vivo CRISPR/Cas9 genome editing therapy, NTLA-2001,” says the company.
“The ability to re-dose is a key advantage of Intellia’s non-viral, lipid nanoparticle (LNP)-based delivery platform. These results are the first-ever clinical data demonstrating redosing with a CRISPR-based medicine, enabling the potential treatment of diseases where a target additive pharmacodynamic effect is desired,” they added.
NTLA also jumped on news that data from a Phase 1 study of NTLA-2002 for hereditary angioedema, or HAE was positive. As noted by the company, a single dose of NTLA-2002 led to a 98% mean reduction in the monthly heart attack rate. It also led to a 99% average reduction in moderate to severe attacks.
“These unprecedented data strengthen our view that NTLA-2002 could be a groundbreaking treatment for people living with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D.
Editas Medicine (EDIT)
There’s also Editas Medicine (NASDAQ: EDIT), which last traded just under $5 a share.
After falling on poor earnings, JPMorgan analysts upgraded the EDIT stock to an underweight rating, with a $7 price target. “Editas has
a systemic, modular approach that may allow for differentiation over competitors,” they added, as quoted by Seeking Alpha.
Morgan Stanley also upgraded the stock to equal weight with a price target of $7.
Helping, EDIT just announced new safety and efficacy data in 18 patients living with sickle cell disease (SCD) treated with renizgamglogene autogedtemcel (reni-cel). They noted that “In the RUBY trial to date, reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all patients.”
It also announced new safety and efficacy data in 7 patients with transfusion-dependent beta thalassemia (TDT) treated with renizgamglogene autogedtemcel (reni-cel). It was also found to be “well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all patients,” added the company.
On the date of publication, Ian Cooper did not hold (either directly or indirectly) any positions in the securities mentioned. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.