CTX310 společnosti CRISPR Therapeutics ve studii fáze 1a při nejvyšší dávce snížil hladinu triglyceridů v průměru o 48 % a hladinu LDL cholesterolu o 53 %.
CRISPR Therapeutics (CRSP -1.20%) has a major claim to fame. It developed a medicine called Casgevy, which in 2023 became the first approved therapy to use the Nobel Prize-winning CRISPR gene-editing technique. Casgevy is currently CRISPR Therapeutics' only approved medicine, and it shares the rights to it with Vertex Pharmaceuticals (VRTX -2.10%). This therapy is important to CRISPR Therapeutics' prospects, but the company arguably has even more critical catalysts on the horizon.
Notably, the mid-cap biotech has several promising candidates in the pipeline that could make significant progress over the next few years. Let's discuss one of them: CTX310. Here's why this investigational medicine is, perhaps, even more important to CRISPR Therapeutics' future.
Image source: The Motley Fool.
A massive potential addressable market
Casgevy was no doubt a breakthrough, but its commercial opportunity is fairly limited. CRISPR Therapeutics and Vertex Pharmaceuticals have noted that the medicine currently has an addressable market of roughly 60,000 patients across several countries where it is approved, including the U.S., Canada, and several European and Middle Eastern countries.
But CRISPR Therapeutics' CTX310 -- which also utilizes the CRISPR mechanism -- could have a much larger market. This medicine is being designed to decrease LDL cholesterol and triglycerides (TGs), which, at high levels, can cause severe, sometimes life-threatening, cardiovascular and metabolic problems. So, it could reduce the risk of cardiovascular disease in eligible patients. There are approved medications to help patients lower TGs and LDL, most of which are taken once or twice daily.
Nothing wrong with that, but a one-time therapy that addresses this problem once and for all could be a significantly more convenient option than current standards of care. That's what CTX310 could be. What's the addressable opportunity for this medicine? According to CRISPR Therapeutics, more than 40 million patients in the U.S. alone have high LDL or TG levels.
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So, this medicine's commercial opportunity could be far larger than Casgevy's for the biotech, especially since CRISPR Therapeutics is developing it alone and won't have to share its profits with another drugmaker, as it does with Casgevy. CTX310 has, so far, posted encouraging results in clinical trials. In August, CRISPR Therapeutics reported data from a Phase 1a clinical trial in patients with high LDL or TG levels associated with certain genetic conditions. The medicine showed a mean TG reduction of 48% and a mean LDL reduction of 53% at the highest dose.
CRISPR Therapeutics will report data from a Phase 1b study in early November. There is still a long way before this therapy can hope to earn approval, but it is important to CRISPR Therapeutics' prospects, so it will be critical to monitor its clinical progress.
Is CRISPR Therapeutics' stock a buy?
CRISPR Therapeutics has underperformed the broader equity market over the past five years. But the company may bounce back in the medium term, provided it can make significant progress in its pipeline. The biotech has other candidates besides CTX310. For instance, it is working on a medicine called CTX611, an investigational, long-acting anticoagulant. Further, over the next five years, CRISPR Therapeutics could also make commercial progress with Casgevy, especially since a recent label expansion in patients as young as two added 5,500 people to its addressable market in the U.S.
Naturally, there is the very real risk of clinical and regulatory setbacks. Also, gene-editing medicines aren't exactly mainstream in the healthcare community yet, and there are some roadblocks to mass adoption, including their typically high prices and the fact that they are often very difficult to administer. All of these are risks worth factoring in. With all that said, is the stock a buy?
My view is that CRISPR Therapeutics is a fairly attractive biotech company, given its deep gene-editing pipeline that features several potential breakthrough medicines, but only for investors comfortable with significant risk and volatility. The stock might soar through 2030 if it can advance key pipeline programs and avoid commercial pitfalls, but certain setbacks might sink the share price. Invest accordingly.
Akcie společnosti CRISPR Therapeutics za poslední měsíc ztratily 10,3 %; Zacks jim udělil hodnocení Rank #4 (prodat), které naznačuje možné zaostávání za trhem v nejbližším období.
CRISPR Therapeutics AG (CRSP - Free Report) is one of the stocks most watched by Zacks.com visitors lately. So, it might be a good idea to review some of the factors that might affect the near-term performance of the stock.
Over the past month, shares of this company have returned -10.3%, compared to the Zacks S&P 500 composite's -1.2% change. During this period, the Zacks Medical - Biomedical and Genetics industry, which CRISPR Therapeutics falls in, has lost 2.4%. The key question now is: What could be the stock's future direction?
While media releases or rumors about a substantial change in a company's business prospects usually make its stock 'trending' and lead to an immediate price change, there are always some fundamental facts that eventually dominate the buy-and-hold decision-making.
Earnings Estimate RevisionsRather than focusing on anything else, we at Zacks prioritize evaluating the change in a company's earnings projection. This is because we believe the fair value for its stock is determined by the present value of its future stream of earnings.
We essentially look at how sell-side analysts covering the stock are revising their earnings estimates to reflect the impact of the latest business trends. And if earnings estimates go up for a company, the fair value for its stock goes up. A higher fair value than the current market price drives investors' interest in buying the stock, leading to its price moving higher. This is why empirical research shows a strong correlation between trends in earnings estimate revisions and near-term stock price movements.
For the current quarter, CRISPR Therapeutics is expected to post a loss of $1.07 per share, indicating a change of +8.6% from the year-ago quarter. The Zacks Consensus Estimate has changed +8.6% over the last 30 days.
The consensus earnings estimate of -$4.48 for the current fiscal year indicates a year-over-year change of +30.8%. This estimate has changed +2.1% over the last 30 days.
For the next fiscal year, the consensus earnings estimate of $3.64 indicates a change of +18.7% from what CRISPR Therapeutics is expected to report a year ago. Over the past month, the estimate has changed -5.5%.
Having a strong externally audited track record, our proprietary stock rating tool, the Zacks Rank, offers a more conclusive picture of a stock's price direction in the near term, since it effectively harnesses the power of earnings estimate revisions. Due to the size of the recent change in the consensus estimate, along with three other factors related to earnings estimates, CRISPR Therapeutics is rated Zacks Rank #4 (Sell).
The chart below shows the evolution of the company's forward 12-month consensus EPS estimate:
12 Month EPS
Projected Revenue GrowthEven though a company's earnings growth is arguably the best indicator of its financial health, nothing much happens if it cannot raise its revenues. It's almost impossible for a company to grow its earnings without growing its revenue for long periods. Therefore, knowing a company's potential revenue growth is crucial.
In the case of CRISPR Therapeutics, the consensus sales estimate of $9.95 million for the current quarter points to a year-over-year change of +1017.4%. The $32.45 million and $141.72 million estimates for the current and next fiscal years indicate changes of +824.5% and +336.7%, respectively.
Last Reported Results and Surprise HistoryCRISPR Therapeutics reported revenues of $10.18 million in the last reported quarter, representing a year-over-year change of +1043.8%. EPS of -$0.94 for the same period compares with -$1.29 a year ago.
Compared to the Zacks Consensus Estimate of $7.42 million, the reported revenues represent a surprise of +37.28%. The EPS surprise was +14.55%.
Over the last four quarters, CRISPR Therapeutics surpassed consensus EPS estimates two times. The company topped consensus revenue estimates just once over this period.
ValuationWithout considering a stock's valuation, no investment decision can be efficient. In predicting a stock's future price performance, it's crucial to determine whether its current price correctly reflects the intrinsic value of the underlying business and the company's growth prospects.
While comparing the current values of a company's valuation multiples, such as price-to-earnings (P/E), price-to-sales (P/S), and price-to-cash flow (P/CF), with its own historical values helps determine whether its stock is fairly valued, overvalued, or undervalued, comparing the company relative to its peers on these parameters gives a good sense of the reasonability of the stock's price.
The Zacks Value Style Score (part of the Zacks Style Scores system), which pays close attention to both traditional and unconventional valuation metrics to grade stocks from A to F (an A is better than a B; a B is better than a C; and so on), is pretty helpful in identifying whether a stock is overvalued, rightly valued, or temporarily undervalued.
CRISPR Therapeutics is graded F on this front, indicating that it is trading at a premium to its peers. Click here to see the values of some of the valuation metrics that have driven this grade.
ConclusionThe facts discussed here and much other information on Zacks.com might help determine whether or not it's worthwhile paying attention to the market buzz about CRISPR Therapeutics. However, its Zacks Rank #4 does suggest that it may underperform the broader market in the near term.
Finanční ředitel společnosti CRISPR Therapeutics Raju Prasad odchází za příležitostmi mimo společnost; s okamžitou platností ho nahradí dosavadní senior viceprezident financí A.J. Silver.
ZUG, Switzerland and BOSTON, Oct. 19, 2026 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today announced that its Chief Financial Officer, Raju Prasad, Ph.D., will be stepping down from the Company to pursue external opportunities, and A.J. Silver, the Company's Senior Vice President of Finance, will succeed Dr. Prasad as Chief Financial Officer. This transition will be effective today.
“Raju has been a trusted partner to me and a wonderful colleague during his time at CRISPR Therapeutics," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "His thoughtful leadership helped build a strong finance organization, strengthen our balance sheet, and deepen our relationships with the investment community. On behalf of everyone at CRISPR Therapeutics, I thank Raju for all he has done and wish him every success in his next role.”
“I am excited for A.J. to assume his new role as CFO,” added Dr. Kulkarni. “Over the past eight years, A.J. has been a key leader across our finance organization, and his deep knowledge of our business combined with his strong operational experience makes him exceptionally well suited to lead us going forward."
"Serving as CFO of CRISPR Therapeutics has been an honor," said Dr. Prasad. "I am grateful to Sam, the Board of Directors of CRISPR Therapeutics, and the Company’s talented team for all we have accomplished together. It has been inspiring to work alongside people so committed to bringing transformative medicines to patients, and I am confident in the Company's future. I have decided this is the right time for me to take the next step in my career, and I look forward to following CRISPR Therapeutics’ continued progress.”
Mr. Silver has served as the Company’s Senior Vice President of Finance since January 2025. Since joining CRISPR Therapeutics in October 2018, he has held roles of increasing responsibility spanning strategic finance, treasury, financial planning and analysis, accounting and reporting, tax, and financial operations. Before joining the Company, Mr. Silver spent more than 10 years at Ernst & Young LLP, where he became a Certified Public Accountant. He holds a Master of Science in Accounting from the University of Connecticut and a B.S. from Boston College with concentrations in finance and accounting.
About CRISPR Therapeutics
CRISPR Therapeutics is a leading biopharmaceutical company focused on developing transformative gene-based medicines for serious human diseases. Founded over a decade ago as an early pioneer in CRISPR/Cas9 gene editing, the Company has evolved from a pioneering research-stage organization into an industry leader, marking a historic milestone with the approval of CASGEVY® (exagamglogene autotemcel [exa-cel]), the world’s first CRISPR-based therapy, for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia. Today, CRISPR Therapeutics is advancing a broad, diversified pipeline spanning hemoglobinopathies, cardiovascular disease, autoimmune disease, oncology, regenerative medicine and rare diseases. The Company is also expanding its gene editing toolkit through SyNTase™ editing, its novel, proprietary platform designed to enable precise, efficient, and scalable gene correction. To accelerate its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit www.crisprtx.com.
CRISPR Therapeutics získala schválení přípravku Casgevy pro TDT a srpkovitou anémii, první schválené genové terapie založené na technologii CRISPR; další růst závisí na klinickém pokroku lékových kandidátů včetně CTX310.
CRISPR Therapeutics (CRSP -4.41%), a gene editing-focused biotech, has made significant clinical and regulatory progress over the past five years. However, over this period, the company has lagged broader equities. CRISPR Therapeutics' shares have declined by about 47% since mid-October 2021, while the S&P 500 has gained almost 73%, as of writing.
Will CRISPR Therapeutics continue its southbound journey through the end of the decade, or does the company have what it takes to bounce back? Let's see where the biotech might be in 2030.
Pipeline progress will be key In 2023 and early 2024, CRISPR Therapeutics earned approval for Casgevy, a gene-editing medicine for transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD), both of which are blood-related disorders. CRISPR Therapeutics collaborated with Vertex Pharmaceuticals (VRTX -1.77%) to develop this therapy. The approval was an important milestone for the company for at least two reasons.
Image source: The Motley Fool.
First, before Casgevy, CRISPR Therapeutics didn't have a single product on the market. This treatment allowed it to transition to a commercial biotech. Second, Casgevy was the first CRISPR-based gene-editing therapy to earn approval. Since most of CRISPR Therapeutics' pipeline candidates are CRISPR-based gene-editing medicines, Casgevy's launch lent more credence to the company's platform. CRISPR Therapeutics hasn't made much significant clinical progress since Casgevy's approval, but the company has several exciting pipeline candidates that investors should keep an eye on.
One of them is CTX310, a medicine being developed to help lower LDL cholesterol and triglycerides (TGs) in some patients. Why is this important? At high levels, LDL and TGs can cause a range of cardiovascular problems. Treatment options do exist, but they often need to be taken daily. CTX310 could be a one-time option to help some patients reduce LDL and TGs. CRISPR Therapeutics recently reported data from a Phase 1 clinical trial in certain patients with high levels of LDL or TGs, in some cases due to rare genetic disorders. The medicine led to significant decreases in both LDL and TGs.
It's still too early to celebrate, but over the next five years, CRISPR Therapeutics could make significant progress with CTX310 and perhaps earn approval, opening up a potentially highly lucrative opportunity.
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CRISPR Therapeutics could also make meaningful progress with zugo-cel, an investigational medicine for several autoimmune diseases and blood cancers. Another pipeline candidate to keep an eye out for is CTX611, an investigational anticoagulant. CTX611 could be administered just twice a year, unlike today's anticoagulants, which are taken regularly. It is also being developed to reduce the risk of significant bleeding associated with many products in this niche. Perhaps the best-case scenario for CRISPR Therapeutics over the next five years would be to earn approval for all these medicines.
Hedge your bets If CRISPR Therapeutics does launch several of its promising therapies by 2030, the company's financial results will improve significantly, and its share price will likely jump. In the meantime, Casgevy's sales could also ramp up. Earlier this year, the therapy earned a label expansion in treating TDT and SCD patients as young as two, which added about 5,500 patients in the U.S. to its target market. That doesn't sound like a lot, but at a price of $2.2 million per treatment course, that's a meaningful expansion.
However, there are significant risks to consider. CRISPR Therapeutics' leading pipeline candidates could fail in clinical trials. It wouldn't be the first time the company has experienced a clinical setback. Also, many gene-editing medicines -- including some that CRISPR Therapeutics is developing -- are one-and-done treatments that are costly to manufacture and administer. So, the economics of these therapies aren't obviously conducive to profits. CRISPR Therapeutics will have to overcome this challenge.
The biotech is, without question, highly innovative, and it may see its shares soar over the medium term on strong clinical and commercial progress. But there is significant risk, too. Only investors comfortable with that should consider initiating a position in the stock.
Cathie Wood během dvou obchodních dnů nakoupila akcie společnosti CRISPR Therapeutics za 3,4 milionu USD. Tržby přípravku Casgevy ve druhém čtvrtletí meziročně vzrostly o 151 % na 76 milionů USD.
Cathie Wood, the founder of Ark Invest, holds $379.1 million worth of shares of CRISPR Therapeutics (CRSP -4.41%) and on Friday, Oct. 9, and Monday, Oct. 12, went on a CRISPR Therapeutics buying spree, buying $1.1 million worth of the stock and $2.3 million worth of CRISPR stock on back-to-back market days, both in her ARK Genomic Revolution ETF (ARKG -5.16%).
That's a lot of faith in a biotech stock that is down a little more than 25% over the past year.
CRISPR's lab has already demonstrated its CRISPR gene-editing proof of concept when it teamed with Vertex Pharmaceuticals (VRTX -1.77%) to produce Casgevy, which, with one intravenous (IV) infusion, has the potential to cure severe sickle cell disease and beta-thalassemia (two rare genetic blood diseases) in an individual.
Image source: Getty Images.
The problem is that Casgevy is CRISPR's only approved therapy, and it is prohibitively expensive, at a list price of $2.2 million per patient. It also affects a relatively small pool of SCD patients, about 100,000 in the U.S., and fewer than 3,000 beta-thalassemia patients in the U.S., and CRISPR has to share its Casgevy revenue with Vertex, which gets 60% of the revenue.
Despite that, here are three reasons why Wood might be bullish on the gene-editing stock.
1. Improving finances as Casgevy's reach widens
In the second quarter, CRISPR reported $76 million in Casgevy revenue, a rise of 151% year over year. The company still isn't profitable, but its net loss was $91.2 million, down from $208.5 million in the same period a year ago. The company has $2.36 billion in cash, easily enough to last four years, enough time for the company to develop other therapies.
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Those numbers should continue to improve as Casgevy received the nod from the Food and Drug Administration (FDA) in July for use in patients as young as 2 years old with SCD or beta-thalassemia.
2. The next big thing for CRISPR?
Now, however, the company has another promising therapy with a much wider application, CTX-310. It uses small fat-based particles to deliver its gene-editing mechanism to the liver. Once there, it switches off a gene, angiopoietin-like protein 3 (ANGPTL3). Turning off this gene lowers LDL cholesterol and triglycerides, two lipids associated with heart disease.
In a phase 1a trial, CTX-310 demonstrated strong efficacy in reducing LDL cholesterol and triglycerides. Participants who were injected with CTX-310 showed, after 12 months, an average of a 79% reduction in the ANGPTL3 gene, a 53% drop in LDL, and a 48% decline in triglycerides.
3. Another key readout is on the way
The company is in the midst of a phase 2 trial for CTX611, a blood thinner being compared with the current standard, enoxaparin, in patients undergoing knee replacement. It expects a readout on the trial later this year. The therapy works by editing a patient's small interfering RNA (siRNA) to make less of a protein, Factor XI, which forms blood clots.
CTX611 targets a multi-billion-dollar market opportunity across a wide range of clotting and thromboembolic conditions. Key potential indications include atrial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-associated thrombosis (CAT), peripheral vascular disease (PVD), chronic coronary artery disease (CAD), and thrombosis in chronic kidney disease (CKD).
Citi zvýšila cílovou cenu společnosti CRISPR Therapeutics na 88 USD, což představuje potenciál růstu o 60 % díky rostoucí poptávce po genové terapii Casgevy.
It's been a disappointing past few years for patient shareholders of CRISPR Therapeutics (CRSP +4.87%). Despite the company passing multiple major milestones during this stretch, this biopharma/biotechnology stock's gone nowhere since falling back from its early 2021 peak.
Namely, in late 2023, the U.S. Food and Drug Administration approved CRISPR's Casgevy as a treatment for sickle cell disease, making it one of the nation's very first gene-editing-based therapies approved for any use. Patient uptake began shortly thereafter.
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Analysts aren't deterred though, including Citi's Yigal Nochomovitz, who recently raised his price target on CRSPR from $82 to $88 per share on growing demand for the aforementioned Casgevy. That's 60% above the stock's present price.
The thing is, what needs to happen to pump this stock's price up to that lofty level is certainly possible. Here's a closer look.
It's in the name CRISPR Therapeutics' claim to fame -- as well as its drug pipeline and portfolio -- is rooted in the ability to repair damaged DNA by replacing it with a correct sequence of genetic information -- a discovery for which co-founder Emmanuelle Charpentier was awarded a Nobel Prize in Chemistry in 2020.
The underlying science is in the name, in fact. CRISPR -- an acronym for "clustered regularly interspaced short palindromic repeats" -- is a genetic sequence that can be identified and then spliced into by an RNA-guided DNA endonuclease protein called Cas9, allowing for the replacement of faulty genetic code in a strand of DNA.
Although the company's initial target was the relatively low-hanging fruit of sickle cell disease, the idea holds promise as a treatment for any number of genetics-driven disease. To this end, the company is working on several other therapies.
Right now, however, the market's pricing CRSPR shares based on a relatively small but important number of inputs.
Three things that must go right for Citi's CRISPR Therapeutics price target It's not all that matters, to be clear. But, for the time being anyway, investors are expectantly looking at these three factors.
1. Reported Casgevy revenue must be satisfactory CRISPR Therapeutics hasn't reported much Casgevy revenue yet, but it is accruing. It's just going to take time for it to make it to the company's top line in earnest. That's because Casgevy treatments are complex, and it can take a long while to administer and complete a treatment regimen.
Indeed, since each patient's treatment is custom-created from a sample of their own blood, it can take up to a year to deliver the final dosing administered at an authorized treatment center operated by CRISPR Therapeutics' treatment partner, Vertex Pharmaceuticals (VRTX +0.82%). Never even mind the length of time it might take an insurer to authorize the $2.2 million cost per Casgevy treatment. And even beyond that, any payments and insurance reimbursements are first processed by Vertex, which takes a cut.
Meaningful revenue is coming, though. In its second-quarter investor update posted in early August, CRISPR Therapeutics confirmed that the custom-created drug generated $76 million in total revenue over three months, a chunk of which is eventually headed to CRISPR's actual top line.
The exact amounts and time frames are still in question; both are moving targets. For what it's worth, though, the analyst community is looking for this year's collaboration and grant-led top line of $40.8 million to soar to $156.6 million next year, once Casgevy is finally driving real, tangible revenue for the company. Anything that puts the company anywhere on track for anything near that projected 2027 number will be just fine.
2. Encouraging progress from CTX310 Casgevy isn't the only drug under CRISPR's umbrella, even if it's the only one approved and on the market right now. CTX340 is a promising answer to refractory hypertension, for instance, while CTX460 showed efficacy as a potential treatment for problematic liver proteins, a condition known as alpha-1 antitrypsin deficiency, or AATD.
Image source: Getty Images.
Perhaps the one drug currently in the company's developmental pipeline with the potential to really move the needle, though, is CTX310, for the treatment of the cardiovascular disease linked to liver-made proteins that help regulate blood lipid levels. Using the same gene-editing science that helped create Casgevy, CRISPR Therapeutics hopes the treatment will help reduce patients' genetically high cholesterol.
Although no specific update date has been set, CRISPR reported in August that it expects to post an update of CTX310's phase 1 clinical trials currently underway sometime before the end of the year. Given the obvious need for such a treatment (the global lipid-lowering drug market will be worth nearly $50 billion per year by 2035, according to Precedence Research), encouraging developmental progress on this front could help drive shares toward Citi's recently raised price target.
3. Proving a drug like zugo-cel can work Finally, while an update on CTX310's progress could help make or break CRSPR stock in the foreseeable future, it's not the only drug in CRISPR Therapeutics' pipeline with the potential to spark bullish interest in this ticker. A chimeric antigen receptor T-cell-based investigational cancer therapy called zugocabtagene geleucel -- or Zugo-cel, for short (formerly CTX112) -- is a prospective game changer for the company as well. Just not for the reason you might think.
While effective oncology drugs' value is clear, that's not what makes zugo-cel so interesting. Rather, what merits attention here is the fact that this therapy is an allogeneic treatment. That just means while it's technically a gene-editing drug, it doesn't require a patient-specific sample to create a custom-built therapy. It simply needs a healthy starting sample from anyone to turn into a medicine that can be administered to everyone. This simpler process costs dramatically less to utilize than Casgevy's patient-specific treatment approach.
It's not just for cancer either. Although the company's using zugo-cel to address hematologic malignancies, it may hold even more promise as a treatment for a range of autoimmune diseases, including lupus, anemia, and systemic sclerosis.
The resulting upshot for the stock will largely depend on how well the market understands this allogeneic gene therapy approach is more flexible and cost-effective than Casgevy's ex-vivo approach that requires the extraction of a specific patient's stem cells.
ZUG, Switzerland and BOSTON, Oct. 08, 2026 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today announced that clinical data from the Phase 1b trial of CTX310®, its investigational CRISPR/Cas9 in vivo gene editing therapy targeting angiopoietin-related protein 3 (ANGPTL3), in patients with severe hypertriglyceridemia will be presented in a late-breaking oral presentation at the American Heart Association (AHA) Scientific Sessions 2026, taking place November 6 – 9, 2026.
Abstract Title: CRISPR-Cas9 Gene Editing Therapy Targeting ANGPTL3 for Severe Hypertriglyceridemia: Results From Phase 1b Trial
Session Type: Late-Breaking Science (Oral Presentation)
Session Title: LBS.05 New Frontiers in Cardiometabolic Risk Reduction
Session Date and Time: Monday, November 9, 2026, 8:00 a.m. CST
The accepted abstract titles are available online on the AHA website. The data in the abstract and presentation are embargoed until the date and time of presentation. A copy of the presentation will be available at www.crisprtx.com once the presentation concludes.
About In Vivo Liver Editing Programs
CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase™ editing technology. The Company's in vivo portfolio includes three cardiovascular programs: CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), in development for heterozygous and homozygous familial hypercholesterolemia, mixed dyslipidemias, and severe hypertriglyceridemia; CTX340™, targeting angiotensinogen (AGT), in development for refractory hypertension; and CTX321™, targeting LPA, in development for patients with elevated lipoprotein(a) [Lp(a)]. In addition, the Company's disclosed development candidates also include CTX460™, targeting SERPINA1 using SyNTase editing, for the treatment of alpha-1 antitrypsin deficiency (AATD).
About CRISPR Therapeutics
CRISPR Therapeutics is a leading biopharmaceutical company focused on developing transformative gene-based medicines for serious human diseases. Founded over a decade ago as an early pioneer in CRISPR/Cas9 gene editing, the Company has evolved from a pioneering research-stage organization into an industry leader, marking a historic milestone with the approval of CASGEVY® (exagamglogene autotemcel [exa-cel]), the world’s first CRISPR-based therapy, for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia. Today, CRISPR Therapeutics is advancing a broad, diversified pipeline spanning hemoglobinopathies, cardiovascular disease, autoimmune disease, oncology, regenerative medicine and rare diseases. The Company is also expanding its gene editing toolkit through SyNTase™ editing, its novel, proprietary platform designed to enable precise, efficient, and scalable gene correction. To accelerate its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit www.crisprtx.com.
CRISPR THERAPEUTICS® standard character mark and design logo, CTX310®, CTX321™, CTX340™, CTX460™ and SyNTase™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners.
CRISPR Therapeutics Forward-Looking Statement
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials and expectations regarding data, safety and efficacy generally; (ii) data included in the above-described oral presentation and any associated abstract; and (iii) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9, as well as other technologies. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in CRISPR Therapeutics most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.
CRISPR Therapeutics čekají do konce roku 2026 dvě klíčová data: CTX611 ve fázi 2 a CTX310 u pacientů se závažnou hypertriglyceridémií. Trh ale může mít už nyní vysoká očekávání.
Proving a technology works and proving it can win a share of a crowded market are two different things. CRISPR Therapeutics (CRSP -0.26%) will soon be put to the test on both.
The biotech expects to report data for two clinical trial readouts in the coming months. For the stock to do well, the market will need to be impressed by the data, not merely have its (high) expectations confirmed. Here's what to expect.
Image source: The Motley Fool.
This RNA drug is aiming at a crowded market The first catalyst on deck for CRISPR Therapeutics stems from its CTX611 program.
CTX611 is a small interfering RNA (siRNA). It prompts a patient's body to make less of a protein called Factor XI (FXI), which is responsible for helping to form blood clots. Many medicines for that purpose exist, but the candidate's edge is that it might only need to be given two times per year, while most blood thinners on the market right now are formulated as daily pills.
An update from its Phase 2 trial is expected before the end of 2026. That study compares three dose levels of CTX611 against enoxaparin, a standard injectable blood thinner, in patients having knee replacement surgery. The precedent to know here is that in a 2021 trial of another FXI-blocking candidate called abelacimab, clots appeared in 4% of knee surgery patients on the top dose, compared to 22% of those treated with enoxaparin.
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So CRISPR's trial is already calibrated to have good odds of one-upping the standard of care, and it won't be the only candidate in development to do so if it manages to clear that bar.
Therefore, to wow the market and create a strong boost in the company's share price from the readout catalyst, CTX611 will likely need to perform better than both the comparator from its trial (enoxaparin) and other programs in development by rival biopharmas with different modalities.
The second catalyst is subject to the same dynamic The second catalyst for CRISPR is another data update due before the end of the year, this time for CTX310, an infusion that edits the genomes of liver cells to disrupt the gene for ANGPTL3.
That gene codes for a protein that reduces the rate at which fat-carrying particles are cleared from the patient's blood. Disabling it aims to decrease both the patient's level of triglycerides and their level of low-density lipoprotein (LDL) cholesterol -- both of which would be highly lucrative.
In a Phase 1a trial of CTX310, one year after treatment, patients who received the maximum tested dose saw their triglycerides fall by an average of 48%, and their LDL by 53%. The data update coming out is centered around people with severe hypertriglyceridemia (sHTG), whose very high triglycerides raise the risk of acute pancreatitis.
But CRISPR might have the same issue with CTX310 as it does with CTX611.
Arrowhead Pharmaceuticals reported that its drug plozasiran, which is given four times per year and is already approved for a rare, inherited form of the disease, reduced patients' median triglycerides by about 80% in two Phase 3 trials. It plans to apply for U.S. Food and Drug Administration (FDA) approval in sHTG before the end of 2026.
Different trials with different treatment modalities can't be compared rigorously. Still, a gene editing therapy like CTX310 can't be undone once a patient has been dosed, and the bar for it to clear is a drug that doctors can simply stop prescribing at any time if necessary. That's before even getting into the stark differences in efficacy or potential side effects.
So, is the market underestimating these catalysts? Probably not by much.
If CRISPR ends up publishing some stunning and positive data that there was no hint of in its prior releases, the stock could still go on a tear, but don't hold your breath. The competing candidates appear to be strong, and they might be viewed as less risky for patients too.
CRISPR Therapeutics owns the only approved gene therapy of its kind, yet the stock sits far below where Wall Street thinks it belongs. One analyst sees a path to gains that would embarrass the broader market, but the numbers tell…
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CRISPR Therapeutics (NASDAQ:CRSP | CRSP Price Prediction) trades at $59.03, while the average Wall Street price target sits at $87.56. That leaves the stock roughly 48% below where analyst consensus thinks it belongs.
The Swiss-domiciled biotech co-developed CASGEVY with Vertex (NASDAQ:VRTX), the first CRISPR-based gene therapy ever cleared for commercial use. It treats sickle cell disease and transfusion-dependent beta thalassemia and is now approved in 39 countries. Partner-reported CASGEVY revenue jumped 151% year over year in Q2 to $76M.
Commercial progress has not translated to the stock. One Piper Sandler analyst carries a Street-high $110 target, implying roughly 86% upside from here.
Twelve Months of Sideways Grinding on Weak Revenue CRSP is down 4.94% over the past year while the S&P 500 climbed 15.98%. The damage came from steady drift rather than any single blowup. Full-year 2025 revenue was $3.51M, and Q4 2025 revenue collapsed 97.5% year over year to $864K as Vertex’s cost-deferral arrangement lapsed and collaboration expense spiked to $53.7M. Q1 2026 revenue then missed by 65.73%.
Cash burn was heavy, with a 2025 net loss of $581.6M. Even after Q2’s 37% revenue beat, the market has treated CRSP as a story stock in limbo.
Piper Sandler’s $110 Anchors the Bull Case The most aggressive published call belongs to Edward Tenthoff at Piper Sandler, who reiterated Overweight and lifted his target to $110 after CRISPR closed a $600 million convertible note offering that pushed pro forma cash to roughly $2.56 billion. That balance sheet fully funds R&D for years without dilutive equity raises.
Tenthoff models 2026 CASGEVY revenue around $300 million on roughly 150 patient starts, with FDA clearance for children ages 5-11 opening a fresh cohort via priority review vouchers. Consensus is nowhere near that bullish; the Street’s 2026 revenue estimate sits at just $40.83M, climbing to $156.58M in 2027. Any acceleration in uptake could trigger sharp revisions.
The wholly-owned pipeline is the wild card, with CTX310 Phase 1b durability data due at the ESC Congress and zugo-cel autoimmune and B-cell lymphoma readouts pending.
EPS revisions have skewed upward, with 12 upward revisions in the trailing 30 days versus 3 down for FY2026.
Every Gene-Editing Name Sits at a Steep Discount The whole cohort is dislocated, and CRISPR is actually the least discounted of the group. Intellia Therapeutics (NASDAQ:NTLA) trades at $12.69 versus a $24.00 consensus target for about 89% implied upside, though shares are down 26.48% over the past year.
Beam Therapeutics (NASDAQ:BEAM) sits at $25.93 against a $52.15 average target, roughly 101% upside, with analysts almost unanimously Buy-rated (13 of 15). Editas Medicine (NASDAQ:EDIT) is a $2.92 stub with a $6.00 target for roughly 105% implied upside, though 2 Strong Sells against 6 Buys signal real skepticism.
Analyst targets are not guarantees anywhere in this space. While EDIT shows the largest implied gap on paper, CRSP is the only name in this group with an approved commercial product generating partner-reported revenue.
Consensus Says 48%, Piper Says 86% CRSP trades at $59.03 with a $5.71B market cap. The consensus $87.56 target across 22 covering analysts implies about 48% upside; Piper Sandler’s Street-high $110 implies roughly 86%.
Shares are up 13.89% on the week, up 12.57% year to date, and down 4.94% over the past year. The S&P 500 is up 13.41% YTD and 15.98% over the past year.
Where I Land on CRISPR CRISPR Therapeutics looks compelling here if you believe CASGEVY’s commercial ramp is inevitable and the $2.36B cash cushion gives the in vivo pipeline time to prove itself. But the setup weakens if consensus 2026 revenue of $40.83M already feels generous and you expect pipeline readouts to disappoint.
Bull path: CASGEVY accelerates via pediatric expansion, CTX310 delivers durable Lp(a) reductions, and the Street revises upward toward Piper’s number. Bear path: partner-recognized revenue stays lumpy, gene editing remains out of favor, and cash-burning biotechs stay in the penalty box.
I lean cautiously constructive. CRSP is the only company in this peer group with an approved product on the market, and it trades at less than half of the Street-high target. That risk-reward looks favorable to me, provided you can stomach the volatility.
Contact [email protected] for any questions or corrections.
CRISPR Therapeutics zatím neúčtuje výnosy z Casgevy, ale ve 2. čtvrtletí tento lék technicky přinesl 76 milionů USD, meziročně o 78 % více. Firma navíc čeká další růst díky pipeline a cíleným studiím.
It's been a disappointing couple of years for shareholders of biotechnology outfit CRISPR Therapeutics (CRSP -1.33%). This stock's barely up since the end of 2022, lagging the broad market's gains. It's not the performance that investors keeping tabs on this company were expecting, given its potential.
Don't be discouraged, though. While still speculative like most young biotech names, CRISPR Therapeutics remains a compelling prospect for investors who can stomach the risk and its inevitable volatility. Here's why.
On the off-chance you're reading this and aren't already familiar with the company, CRISPR Therapeutics is a drug developer that specializes in gene editing.
Image source: Getty Images.
It's probably an area you've heard a great deal about. No gene-editing drugs were actually on the market in the United States until CRISPR Therapeutics' Casgevy was approved by the FDA as a treatment for sickle cell disease in late 2023. Any and all gene therapies currently on the market followed this pioneer's foray. Being first is a well-deserved accolade for the company, too, which was co-founded by Dr. Emmanuelle Charpentier, who was one of the co-discoverers of the CRISPR/Cas9 gene-editing mechanism that makes Casgevy work.
CRISPR -- short for "clustered regularly interspaced short palindromic repeats" -- is a specific sequence on a strand of DNA that can be spliced into with a protein called Cas9, allowing it to be replaced with a genetically correct sequence. Charpentier's work, along with Jennifer Doudna's, was so pivotal that the duo won the Nobel Prize in Chemistry in 2020. CRISPR Therapeutics had already done a great deal of developmental work by then, however, recognizing the potential of being able to "fix" damaged genetic code, treating many diseases at the root of their problem.
And at times, this stock performed as brilliantly as you might expect it to ... particularly when the company's co-founder won a Nobel Prize for her work. Other times (and since then), not so much, including this year's lethargic performance.
Just don't give up hope yet. The best is yet to come.
3 reasons CRSP stock is still a buy There's still risk here to be sure. That's just the nature of all biotech stocks, and pre-profit, pre-revenue companies in particular.
There are also reasons to be cautiously bullish, though, three of which stand out from the rest.
1. Revenue is coming Despite the drug being approved all the way back in 2023, it's hard to ignore that CRISPR Therapeutics hasn't officially reported any Casgevy revenue. Don't misunderstand, though. The sickle cell treatment is being administered and accruing revenue. The company just can't book any of it yet.
The holdup is the time required to complete a treatment regimen. Since each treatment is custom-created for that particular patient, the entire process can take up to a year to complete. That's when revenue finally gets officially booked.
It's coming, though. As the company's second-quarter report points out, the drug technically accrued $76 million worth of revenue in Q2, up 78% year over year. Even after treatment partner Vertex Pharmaceutical (VRTX -1.55%) takes its cut, analysts expect CRISPR Therapeutics to report sales of $156 million next year, well up from this year's $41 million in (mostly grant and developmental) revenue, en route to a top line of more than $600 million in 2028.
Even just a hint of this progress could spark some bullishness from this stock.
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2. Its pipeline is underappreciated Largely lost in all the hullabaloo surrounding Casgvey is the fact that CRISPR Therapeutics is working on so much more. Its research and development pipeline currently includes clinical trials of oncology drugs, cardiovascular disease treatments, and others. For instance, its CTX310 is particularly promising as a means of lowering cholesterol. Although still only in phase 1 clinical trials, the in-vivo CRISPR/Cas9 gene editing therapy targeting ANGPTL3 (the liver-made Angiopoietin-like protein 3) that stifles fat-reducing enzymes showed reductions of between 50% and 80% of this protein, depending on the dose.
And another drug, only known as CTX611, is demonstrating strong efficacy as a treatment for thromboembolic and cardiovascular indications, with pivotal phase 2 trial updates anticipated before the end of this year, along with a handful of other developmental updates.
These updates could rekindle bullish sentiment simply by reminding investors that the company is working on profit centers other than Casgevy.
3. It's a drug development platform with enormous potential Finally, look beyond Casgevy, CTX310, and CTX611 at the underlying biotechnology itself. CRISPR Therapeutics' well-patented CRISPR/Cas9 approach to editing genetic code has nearly unlimited possibilities as a drug development platform. It's just a matter of determining which opportunities are most promising and putting its $2.3 billion worth of accessible liquidity to work.
To this end, Precedence Research believes the global CRISPR-based gene-editing drug market is poised to grow at an average annual rate of 12.9% through 2035, reaching nearly $15 billion. Given that the company was co-founded by the geneticist who won a Nobel Prize for this very science, CRISPR Therapeutics is obviously well-positioned to capture at least its fair share of this growth.
It could also be an acquisition target, though that alone is never a sufficient reason to buy or hold a speculative stock. In this instance, there are more than enough other reasons.
CRISPR Therapeutics očekává do konce roku 2026 data z CTX310 pro těžkou hypertriglyceridemii i top-line data fáze 2 z CTX611, zatímco CASGEVY dál roste po rozšíření indikace u dětí.
3 Biotech Stocks That Could Benefit from the Patent CliffCRISPR Therapeutics NASDAQ: CRSP CEO Sam Kulkarni outlined the company’s commercial and clinical priorities during a Morgan Stanley discussion, highlighting continued adoption of CASGEVY and expected data updates across several pipeline programs by the end of 2026.
Kulkarni said the company’s strategy rests on several layers of potential value: the commercial launch of CASGEVY, three later-stage assets that could be positioned for pivotal studies next year, early clinical programs, and research efforts in areas including in vivo cell editing.
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CASGEVY launch and pediatric expansion CRISPR Therapeutics Gains After Earnings as Pipeline Hope GrowsCASGEVY, which CRISPR Therapeutics markets with Vertex Pharmaceuticals for sickle cell disease and transfusion-dependent beta thalassemia, recently received an FDA label expansion for patients ages 2 years and older. Kulkarni described the pediatric indication as a “great tailwind,” citing Vertex’s most recently disclosed quarterly CASGEVY revenue of approximately $76 million and more than 100 patient initiations per quarter.
He said the pediatric population could support additional treatment starts because more children’s hospitals have experience with the required procedures and because earlier intervention may help patients avoid disease-related organ damage.
CRSPR Stock Could Be Ready to Deliver on Its Massive PromiseAccording to Kulkarni, many initial launch bottlenecks—including reimbursement processes, hospital contracting and manufacturing capacity—have largely been addressed. However, the treatment journey can still take six to nine months or longer from initiation to treatment. He said delays are not primarily related to manufacturing, but can reflect exchange transfusions before cell collection and patient scheduling needs.
Kulkarni also pointed to potential gentler conditioning regimens as a future growth driver. He said a regimen that could reduce hospital stays to roughly three days or less, compared with two or three weeks, could broaden access among more moderate patients. He added that CRISPR Therapeutics is pursuing in vivo hematopoietic stem cell editing, which he said could ultimately expand treatment beyond the current markets in the U.S. and Western Europe.
CTX310 data expected in severe hypertriglyceridemia The company expects to report Phase 1b data for CTX310 in severe hypertriglyceridemia by year-end. CTX310 is designed as a one-time CRISPR-Cas9 treatment targeting ANGPTL3, a protein associated with lipid metabolism and cardiovascular risk.
Kulkarni said data presented at the European Society of Cardiology meeting showed reductions of up to 80% in ANGPTL3 at higher dose levels, along with reductions of nearly 50% in LDL cholesterol and triglycerides in a mixed Phase 1a population. He said the company observed that ANGPTL3 reductions remained at approximately 80% one year after treatment in the data presented.
The Phase 1b study is evaluating more defined patient populations, including severe hypertriglyceridemia, mixed dyslipidemia and refractory hypercholesterolemia. Kulkarni said the immediate focus is severe hypertriglyceridemia, where CRISPR Therapeutics aims to assess triglyceride reduction and additional biomarkers such as LDL cholesterol, hemoglobin A1C and hepatic fat fraction.
He also said ANGPTL3 may be complementary to PCSK9 therapies, citing Phase 1a patients who were already receiving PCSK9 treatment and experienced additional LDL reductions. The company plans to use the Phase 1b results to discuss potential pivotal trial designs with regulators.
Factor XI and autoimmune programs CRISPR Therapeutics also expects year-end top-line Phase 2 data for CTX611, an siRNA therapy targeting Factor XI that is being developed with Sirius Therapeutics. The ongoing study is in patients undergoing total knee arthroplasty and compares CTX611 with enoxaparin.
Kulkarni said the program seeks to provide anticoagulation with less bleeding risk than existing direct oral anticoagulants. He said prior Phase 1 studies showed up to 95% reduction in Factor XI. The company expects the Phase 2 update to include high-level comparisons of venous thromboembolism rates between treatment arms, while more detailed bleeding information is expected later.
Potential future settings discussed included secondary stroke prevention, atrial fibrillation patients unable to use direct oral anticoagulants, cancer-associated thrombosis and surgical applications. CRISPR Therapeutics is responsible for Phase 3 development and potential commercialization of CTX611, according to Kulkarni.
For zugo-cel, its allogeneic CAR-T program, the company is prioritizing B-cell-driven autoimmune diseases. Kulkarni said CRISPR Therapeutics is conducting a basket trial across indications that include systemic sclerosis and myositis, with additional data expected by year-end. The company expects those results to help determine whether one or more indications could advance toward pivotal development.
Earlier pipeline and technology priorities Kulkarni said clinical data from the company’s alpha-1 antitrypsin deficiency program and CTX340 for refractory hypertension are expected in 2027. CTX340 targets angiotensinogen, and Kulkarni said changes in blood pressure could potentially be observed within days after gene editing.
Beyond individual programs, he said CRISPR Therapeutics is working to broaden delivery beyond the liver to areas such as the hematopoietic and immune systems, kidneys, central nervous system and muscle. The company is also pursuing more advanced editing approaches, including whole-gene insertion.
Kulkarni said the company is increasingly using artificial intelligence in regulatory writing, operational activities and product design. He said AI tools are helping the company evaluate mutations, optimize editing strategies and potentially accelerate advancement of programs into the clinic.
About CRISPR Therapeutics (NASDAQ:CRSP)CRISPR Therapeutics AG NASDAQ: CRSP is a biotechnology company focused on developing gene-editing medicines using CRISPR/Cas9 technology. The company's programs are designed to modify disease-associated genes in human cells, with the goal of treating serious conditions across hematology, oncology, autoimmune disease and regenerative medicine.
Its most advanced product is Casgevy (exa-cel), an autologous cell therapy developed in collaboration with Vertex Pharmaceuticals. Casgevy uses CRISPR/Cas9 gene editing to modify a patient's blood stem cells and is approved in several markets for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia.
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected].
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Akcie CRISPR Therapeutics v srpnu posílily téměř o 19 % díky silným výsledkům za 2. čtvrtletí a aktualizaci výhledu. Prodeje Casgevy vzrostly mezikvartálně o 78 % na 76 milionů USD.
Pioneering gene-editing company CRISPR Therapeutics (CRSP -1.81%) was a well-performing biotech stock in August. Its shares were hot in the hot month, zooming almost 19% higher thanks to a solid quarterly earnings report and business update.
A welcome update CRISPR released its second-quarter financials and business update early in the month, on Aug. 3, to be exact. It quoted CEO Samarth Kulkarni as saying that this "reflected strong execution across CRISPR Therapeutics' portfolio and platform" -- and he was not wrong.
Image source: Getty Images.
The biotech's one approved product, the blood disorder treatment Casgevy (developed and marketed in partnership with Vertex Pharmaceuticals), saw a 78% quarter-over-quarter jump in sales to $76 million. Better, during the period, the U.S. Food and Drug Administration (FDA) approved Casgevy's label expansion to cover pediatric patients aged 2 and older.
While financial figures aren't as meaningful for biotechs, with their typically feast-or-famine business models, CRISPR's have been looking good lately.
Second-quarter revenue was boosted significantly by a $10 million upfront payment from a license and collaboration deal from a business partner that hasn't been identified. With that, the company's total top line expanded to nearly $10.2 million from the year-ago tally of $892,000.
I should note here that, under the CRISPR/Vertex partnership agreement, CRISPR does not recognize its share of Casgevy sales as revenue. Rather, its net share is bundled with its proportion of costs in the "collaboration expense, net" line item of its profit and loss statement.
As for CRISPR's bottom line, a steep decline in in-process research and development expenses, plus that $10 million infusion, narrowed the net loss considerably. It was just under $91.5 million ($0.94 per share) for the period compared to the second quarter of 2025's nearly $209 million deficit.
Analysts tracking the stock were modeling revenue of less than $7.5 million and a net loss of $1.20 per share.
CRISPR is a busy company that continues to use its proprietary gene-editing platform to develop new medications. In its pipeline are treatments targeting disorders like hypertension (high blood pressure) and alpha-1 antitrypsin deficiency, a genetic condition that can threaten the lungs and liver.
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Bright future Since we're still near the start of the gene-editing revolution in healthcare, it's likely to be some time before medicines developed with the technology become commonly available. While there are now numerous gene-editing businesses on the scene, CRISPR is a rare bird that has helped bring an actual product to market.
With that, I think it's always going to be in the mix with this future-forward technology, and I'd fully expect more products from its lab to reach pharmacy shelves. This remains a high-potential stock, I believe, but investors need to be patient with it.
FDA schválila CASGEVY od CRISPR Therapeutics, čímž potvrdila platformu CRISPR/Cas9 a posílila investiční atraktivitu firmy. Tržní kapitalizace je 5,6 mld. USD a čistá hotovost 1,78 mld. USD.
SummaryCRISPR Therapeutics transitioned from speculative biotech to commercial stage with FDA approval of CASGEVY, validating its CRISPR/Cas9 platform.CRSP's pipeline targets multi-billion-dollar markets in cardiovascular, autoimmune, oncology, and diabetes, supported by a robust IP portfolio and major pharma partnerships.Current $5.6B market cap and $1.78B net cash position offer acquisition appeal, but most pipeline assets remain early stage and execution risks persist.I view CRSP as a speculative opportunity, with greater interest below $52 where valuation aligns better with balance sheet and pipeline potential. laddawan punna/iStock via Getty Images
Overview CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) gene editing is one of the most important advances in modern biotechnology, allowing scientists to alter specific sections of DNA instead of just treating the downstream symptoms of a
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Analyst’s Disclosure: I/we have a beneficial long position in the shares of CRSP either through stock ownership, options, or other derivatives. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.
I own 100 shares of CRSP with a $39 cost basis.
Seeking Alpha's Disclosure: Past performance is no guarantee of future results. No recommendation or advice is being given as to whether any investment is suitable for a particular investor. Any views or opinions expressed above may not reflect those of Seeking Alpha as a whole. Seeking Alpha is not a licensed securities dealer, broker or US investment adviser or investment bank. Our analysts are third party authors that include both professional investors and individual investors who may not be licensed or certified by any institute or regulatory body.
CRISPR Therapeutics uvedla, že jednorázová infuze CTX310 v rané studii po 1 roce snížila ANGPTL3, triglyceridy i cholesterol LDL. Při nejvyšší dávce klesl ANGPTL3 v průměru o 79 %, triglyceridy o 48 % a LDL o 53 %.
-Data presented in a late-breaking presentation at the European Society of Cardiology (ESC) Congress 2026-
-Phase 1a clinical data for CTX310® continued to demonstrate robust, dose-dependent reductions in circulating ANGPTL3 with a mean reduction from baseline of 79% (maximum 89%), a mean reduction in triglycerides (TG) of 48% (maximum 78%), and a mean reduction of low-density lipoprotein (LDL) of 53% (maximum 84%) at the highest dose-
-CTX310 was well tolerated with no treatment-related serious adverse events, no ≥Grade 3 changes in liver transaminases, and no additional treatment-related adverse events since the previous update-
-Findings simultaneously published in The New England Journal of Medicine entitled “Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310”-
ZUG, Switzerland and BOSTON, Aug. 28, 2026 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today presented durability data from its Phase 1a clinical trial evaluating CTX310®, an investigational, in vivo CRISPR/Cas9 gene editing therapy targeting ANGPTL3. A single-course treatment with CTX310 produced deep and durable reductions in circulating ANGPTL3, triglycerides (TG), and low-density lipoprotein (LDL) that were sustained through one year of follow-up. At the highest dose, mean reductions from baseline were 79% (maximum 89%) for ANGPTL3, 48% (maximum 78%) for TG, and 53% (maximum 84%) for LDL. These extended follow-up data demonstrate the durability of CTX310's effect and support its potential to deliver long-lasting lipid lowering after a single intravenous (IV) infusion.
These data were presented today during a late breaking session at the European Society of Cardiology (ESC) Congress 2026 and extend the Phase 1 results previously presented. The data were also published today in The New England Journal of Medicine (NEJM) in a peer reviewed article entitled "Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310." CRISPR Therapeutics is advancing CTX310 in a Phase 1b clinical trial, with U.S. and ex-U.S. trials ongoing, and expects to provide an additional update on the CTX310 program in the second half of 2026.
"These durability data mark an important next step for the CTX310 program and for the field of in vivo gene editing," said Naimish Patel, M.D., Chief Medical Officer, CRISPR Therapeutics. "Evidence that a single dose can produce lasting lipid lowering is central to our goal of developing one-time treatments for cardiometabolic diseases. These results provide strong support for continued advancement of CTX310 and our broader cardiovascular gene editing portfolio, and we look forward to sharing new data from the severe hypertriglyceridemia cohort of the CTX310 Phase 1b trial in the second half of this year."
"For patients at high cardiovascular risk, the biggest challenge is often not starting therapy but staying on it, since daily medications require lifelong adherence that many patients are unable to maintain," said Luke Laffin, M.D., principal investigator and Medical Director of the Cleveland Clinic Coordinating Center for Clinical Research. "A single infusion producing durable reductions at one-year is an encouraging signal that a one-time approach could help close that adherence gap."
"What is compelling about this update is that the reductions in ANGPTL3, triglycerides, and LDL from a single infusion have persisted out to one-year, suggesting a sustained biological effect," said Steven E. Nissen, M.D., senior author of the study and Chief Academic Officer at the Cleveland Clinic Heart, Vascular and Thoracic Institute. "A one-time treatment with this degree of durability could represent a meaningful advance in how we manage lifelong lipid disorders."
Phase 1a Clinical Trial Design
The Phase 1a portion of the study was an open label, dose-escalation trial evaluating single-course IV doses of CTX310 ranging from 0.1 to 0.8 mg/kg (lean body weight) targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias (elevated TG and LDL). Eligible participants had uncontrolled TG levels >150 mg/dL and/or LDL cholesterol >100 mg/dL (or >70 mg/dL for those with established ASCVD) despite background standard of care per local guidelines.
The majority of participants were receiving statins and/or ezetimibe, while 40% were taking PCSK9 inhibitors. The trial was designed to evaluate safety and tolerability as primary endpoints, with changes in circulating ANGPTL3 protein, TG, and LDL as secondary endpoints.
Safety and Tolerability
Single-course ascending doses of CTX310 were administered to 15 participants across sequential cohorts, and all participants completed at least one-year of follow-up as of the data cutoff. CTX310 was generally well tolerated, and no dose-limiting toxicities or serious adverse events related to treatment. Adverse events were generally mild to moderate.
As previously reported in Laffin et al. (2025), one participant experienced an allergic reaction that resolved the following day with supportive care. Infusion-related reactions occurred in three participants (two at 0.6 mg/kg and one at 0.8 mg/kg dose), all Grade 2. All events resolved, and all participants completed their infusions. Beyond the transient aminotransferases elevation previously reported in one participant shortly after treatment, there were no liver function test elevations throughout the remainder of the trial.
The safety events described above were previously reported in Laffin et al. (New England Journal of Medicine, 2025) and reflect the initial treatment period of the Phase 1a clinical trial. No new treatment-related safety events were observed during extended follow-up.
Efficacy Highlights
These new results build upon previously disclosed clinical data from 15 participants across four sequential cohorts, corresponding to lean body weight-based doses of DL1 [0.1 mg/kg], DL2 [0.3 mg/kg], DL3 [0.6 mg/kg], DL3.5 [0.7 mg/kg] and DL4 [0.8 mg/kg]. All participants had at least one-year of follow-up.
Dose dependent reductions in circulating ANGPTL3 protein were sustained through one-year following CTX310 infusion.Among participants treated at 0.8 mg/kg, ANGPTL3 reductions of up to 89% were observed, with a mean reduction of 79% at one-year following CTX310 infusion. Next Steps
The Phase 1b portion of the trial examines a fixed flat dose regimen of CTX310 equivalent to the most efficacious dose in Phase 1a (0.8mg/kg). CRISPR Therapeutics continues to anticipate sharing an update from the CTX310 Phase 1b clinical trial in the second half of 2026, focused on severe hypertriglyceridemia patients.
About In Vivo Liver Editing Programs
CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase™ editing technology. The Company's in vivo portfolio includes three cardiovascular programs: CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), in development for heterozygous and homozygous familial hypercholesterolemia, mixed dyslipidemias, and severe hypertriglyceridemia; CTX340™, targeting angiotensinogen (AGT), in development for refractory hypertension; and CTX321™, targeting LPA, in development for patients with elevated lipoprotein(a) [Lp(a)]. In addition, the Company's disclosed development candidates also include CTX460™, targeting SERPINA1 using SyNTase editing, for the treatment of alpha-1 antitrypsin deficiency (AATD).
About CRISPR Therapeutics
CRISPR Therapeutics is a leading biopharmaceutical company focused on developing transformative gene-based medicines for serious human diseases. Founded over a decade ago as an early pioneer in CRISPR/Cas9 gene editing, the Company has evolved from a pioneering research-stage organization into an industry leader, marking a historic milestone with the approval of CASGEVY® (exagamglogene autotemcel [exa-cel]), the world’s first CRISPR-based therapy, for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia. Today, CRISPR Therapeutics is advancing a broad, diversified pipeline spanning hemoglobinopathies, cardiovascular disease, autoimmune disease, oncology, regenerative medicine and rare diseases. The Company is also expanding its gene editing toolkit through SyNTase™ editing, its novel, proprietary platform designed to enable precise, efficient, and scalable gene correction. To accelerate its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit www.crisprtx.com.
CRISPR THERAPEUTICS® standard character mark and design logo, CTX310®, CTX321™, CTX340™, CTX460™ and SyNTase™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.
Reference
Laffin L, et al. Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3. The New England Journal of Medicine. 2025.
CRISPR Special Note Regarding Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; (ii) data included in the above-described oral presentation and any associated abstracts or posters, data included in the above-described article in The New England Journal of Medicine, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; and (iii) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9 and SyNTase, as well as other technologies. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in CRISPR Therapeutics most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.
CRISPR Therapeutics AG (CRSP - Free Report) came out with a quarterly loss of $0.94 per share versus the Zacks Consensus Estimate of a loss of $1.1. This compares to a loss of $1.29 per share a year ago. These figures are adjusted for non-recurring items.
This quarterly report represents an earnings surprise of +14.55%. A quarter ago, it was expected that this company would post a loss of $1.14 per share when it actually produced a loss of $1.28, delivering a surprise of -12.28%.
Over the last four quarters, the company has surpassed consensus EPS estimates two times.
CRISPR Therapeutics, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $10.18 million for the quarter ended June 2026, surpassing the Zacks Consensus Estimate by 37.28%. This compares to year-ago revenues of $0.89 million. The company has topped consensus revenue estimates just once over the last four quarters.
The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.
CRISPR Therapeutics shares have lost about 8.5% since the beginning of the year versus the S&P 500's gain of 9.4%.
What's Next for CRISPR Therapeutics?While CRISPR Therapeutics has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?
There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.
Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.
Ahead of this earnings release, the estimate revisions trend for CRISPR Therapeutics was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.
It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is -$1.19 on $7.93 million in revenues for the coming quarter and -$4.90 on $28.88 million in revenues for the current fiscal year.
Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 37% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.
One other stock from the same industry, CAMP4 Therapeutics Corporation (CAMP - Free Report) , is yet to report results for the quarter ended June 2026.
This company is expected to post quarterly loss of $0.24 per share in its upcoming report, which represents a year-over-year change of +61.3%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days.
CAMP4 Therapeutics Corporation's revenues are expected to be $1.35 million, down 10% from the year-ago quarter.
Crispr Therapeutics získala od FDA schválení pro pediatrické použití Casgevy, čímž rozšířila svůj adresovatelný trh. Akcie CRSP po zprávě vyskočily o více než 8 %.
SummaryCrispr Therapeutics secured FDA pediatric approval for Casgevy, expanding its addressable market and reinforcing its leadership in CRISPR/Cas9 gene editing.Casgevy’s robust clinical efficacy, global approvals, and strong safety profile support its multibillion-dollar potential, despite initial slow commercial uptake due to complex treatment and high cost.CRSP’s pipeline includes innovative in-vivo and allogeneic programs targeting cardiovascular and autoimmune diseases, aiming to further simplify and expand gene therapy applications.With pediatric approval, CRSP is positioned for accelerated revenue growth; patient pool expansion and technical advances could drive significant upside toward historic share price highs.Haggerston BioHealth members get exclusive access to our real-world portfolio. See all our investments here » wildpixel/iStock via Getty Images
Investment Overview - Casgevy Secures Pediatric Approval Crispr Therapeutics (CRSP) stock jumped >8% in trading yesterday, reaching a four-month high value of $60 per share and a market cap valuation of $5.79bn, after the company and partner Vertex Pharmaceuticals (
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