Latest news with #CTX320
Associated Press
26-06-2025
- Business
- Associated Press
CRISPR Therapeutics Reports Positive Additional Phase 1 Data for CTX310™ Targeting ANGPTL3 and Provides Update on In Vivo Cardiovascular Pipeline
ZUG, Switzerland and BOSTON, June 26, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases today announced updates across its in vivo cardiovascular disease programs. These include new data for CTX310™, targeting ANGPTL3, as well as continued progress on CTX320™, targeting the LPA gene, and CTX340™, targeting the AGT gene. 'CRISPR Therapeutics remains focused on executing against our strategic priorities and advancing our portfolio of innovative therapies,' said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. 'The additional data from our ongoing Phase 1 clinical trial for CTX310 reinforces the potential of our platform to transform the treatment of serious cardiovascular diseases. We are progressing with our dose-finding study and expect to share complete data at a medical meeting in the second half of this year. For CTX320, we are continuing our dose-finding study and anticipate sharing data in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) treatment landscape.' CTX310, targeting ANGPTL3 CTX320™, targeting LPA CTX340, targeting angiotensinogen (AGT) About In Vivo Programs CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) platform for the delivery of CRISPR/Cas9 to the liver. The Company's in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 ( ANGPTL3 )) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company's research and preclinical development candidates include CTX340 and CTX450™, targeting angiotensinogen ( AGT ) for refractory hypertension and 5'-aminolevulinate synthase 1 ( ALAS1 ) for acute hepatic porphyria (AHP), respectively. About CRISPR Therapeutics Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies 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 CRISPR THERAPEUTICS® standard character mark and design logo CTX310™, CTX320™, CTX340™ and CTX450™ 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 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 made by Dr. Kulkarni in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, status of such studies and trials (including guidance) and expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; (iii) regulatory submissions and authorizations, including related timelines; and (iv) the therapeutic value, development, and commercial potential of gene editing and delivery technologies and therapies, including CRISPR/Cas9. 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 its 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. The Company disclaims 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. This press release also contains information regarding our industry, our business and the markets for certain of our product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Unless otherwise expressly stated, we obtained this industry, business, market and other data from market research firms and other third parties, including medical publications, government data and similar sources. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this Contact: +1-617-307-7503 [email protected] Media Contact: +1-617-315-4493 [email protected]
Yahoo
15-02-2025
- Business
- Yahoo
Why CRISPR Therapeutics (CRSP) Soared on Friday?
We recently published a list of . In this article, we are going to take a look at where CRISPR Therapeutics AG (NASDAQ:CRSP) stands against other firms that soared on Friday. Wall Street finished mixed anew on Friday, as investors continued to weigh fresh developments on tariffs and inflation data. The Nasdaq was the sole index eking out gains, up 0.41 percent. The Dow Jones decreased by 0.37 percent while the S&P 500 was little changed, dipping 0.01 percent. In line with Nasdaq's rally, 10 companies predominantly under the technology sector led the charge. In this article, we detailed the reasons behind their stellar performance. To come up with this list of biggest advancers, we considered only the stocks with at least $2 billion in market capitalization and $5 million in daily trading volume. A laboratory cultivating cells for the development of gene and cell therapies. CRISPR Therapeutics AG (NASDAQ:CRSP) rose for a fourth day on Friday, ending the day higher by 14.33 percent to finish at $49.72 apiece, as investors cheered its upgraded rating from an investment advisory firm. On Friday, Evercore ISI upgraded CSP to 'outperform' from 'in line,' while significantly raising its price target by 65 percent to $99 from $60 previously, on expectations that its clinical trial programs, CTX320 and CTX310, would succeed by 10 percent and 5 percent, respectively, and contribute to the company's growth. CTX320 is in an ongoing clinical trial in patients with elevated lipoprotein(a), a cardiovascular risk factor that affects up to 20 percent of the global population. Dose escalation is ongoing, with an update expected in the first half of 2025. Meanwhile, CTX310 is in an ongoing clinical trial targeting ANGPTL3 in patients with homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (SHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias. Dose escalation is ongoing, with an update expected in the first half of 2025. Overall, CRSP ranks 7th on our list of firms that soared on Friday. While we acknowledge the potential of CRSP as an investment, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than CRSP but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock. READ NEXT: 20 Best AI Stocks To Buy Now and Complete List of 59 AI Companies Under $2 Billion in Market Cap Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
Yahoo
14-02-2025
- Business
- Yahoo
CRISPR Therapeutics Stock Upgrade Following Analyst Reassessment
CRISPR Therapeutics (CRSP, Financials) stock rose 16.4% to $50.60 as of 10:10 a.m. GMT-5 on Friday, following an analyst upgrade. Citing pipeline advancements and possible catalysts, Evercore ISI analyst Liisa Bayko raised CRISpen Therapeutics from "In Line" to "Outperform." The company focused on the CTX320 and CTX310 in vivo gene editing initiatives, giving respectively 10% and 5% success rates. Second quarter of 2025 data from CTX320 is anticipated to line up with Novartis's cardiovascular outcomes study. Mid-2025 should provide updates on CTX112 (CAR-T treatment), CTX131 (oncology), and CTX221 (type 1 diabetes). Warning! GuruFocus has detected 4 Warning Signs with CRSP. From $1.7 billion at the end of 2023, the corporation said in December 31, 2024, $1.9 billion in cash, cash equivalents, and marketable securities. Compared with 40 at the end of October, the Casgevy cell collection program also grew with over 50 fresh collections in the last nine weeks of the fourth quarter. Although the firm's risk-adjusted model puts in low success probability for its new in vivo activities, its financial situation and clinical advancements indicate a better picture. This article first appeared on GuruFocus.
