CRISPR Therapeutics Unveils Promising AATD Gene Editing Data
CRISPR Therapeutics (CRSP) has presented new preclinical data demonstrating that its investigational CTX460, based on the SyNTase gene editing platform, achieved over 90% mRNA correction and a five-fold increase in total serum Alpha-1 Antitrypsin (AAT) levels in animal models of Alpha-1 Antitrypsin Deficiency (AATD).
The CTX460 program is the first candidate from SyNTase and is expected to enter clinical trials by mid-2026. The data were showcased at the 2025 European Society of Gene and Cell Therapy (ESGCT) Annual Congress, highlighting the potential for SyNTase to address the underlying genetic cause of AATD.
While the company faces financial challenges, including negative margins and uncertain profitability, its shares have risen over 40% in the past year, supported by optimistic analyst ratings and a strong pipeline including Casgevy for sickle-cell disease.
Additionally, upcoming regulatory changes by the US FDA to streamline gene editing therapy approvals may accelerate CRISPR Therapeutics' development efforts. The company plans a $600 million share offering aimed at bolstering its financial position amid ongoing research and development investments.
Summary
CRISPR Therapeutics Reports New Preclinical Data From SyNTase Gene Editing Platform For Treatment Of Alpha-1 Antitrypsin Deficiency - CRISPR Therapeutics (NASDAQ:CRSP)
The world's oldest known for its innovative approach to the art of art, the creation of art and science, is now being used as a tool to develop and create new art forms. The company is set to release its own version of this article, which includes a detailed review of the scientific significance of the art behind the art. The article also mentions the potential impact on the future of the world's art and creativity.
Published: October 10, 2025
Published by: benzinga.com
What CRISPR Therapeutics (CRSP)'s Positive CTX460 Gene Editing Data Means for Shareholders
CRISPR Therapeutics (CRSP) has presented new preclinical data at the ESGCT 2025 Annual Congress showing its investigational CTX460, using the SyNTase gene editing platform, achieved greater than 90% mRNA correction and a five-fold increase in total serum AAT in animal models of Alpha-1 Antitrypsin Deficiency (AATD). The results suggest a promising potential for SyNTases to address the underlying genetic cause of AATD, addressing a major unmet medical need for the disease. However, the company's profitability remains uncertain and carries significant risk for shareholders. Despite this, despite retreating from the market, shares are still trading 48% above their fair value.
Published: October 15, 2025
Published by: ca.finance.yahoo.com
CRSP Showcases Promising Preclinical Data for AATD Treatment
CRISPR Therapeutics (CRSP) has reported promising preclinical results for its SyNTase gene editing platform, which targets Alpha-1 Antitrypsin Deficiency (AATD). The company's CTX460 program has shown significant potential in correcting liver DNA and mRNA in animal models. The program's initial program marks the initial investigative use of SyNTASE editing, with clinical trials expected to begin by mid-2026. Despite challenges, the company's financial performance reflects its focus on research and development. Despite these challenges, analyst sentiment remains optimistic, with a recommendation of 2.2, indicating a strong buy-out trend. The company also faces regulatory challenges and potential volatility, which could significantly impact the company’s future trajectory.
Published: October 10, 2025
Published by: gurufocus.com
CRISPR Therapeutics (CRSP) Gains as FDA Eases Approval Pathway
The US FDA's plan to simplify the approval process for gene editing therapies could significantly impact CRISPR Therapeutics (CRSP) and the broader gene editing sector. The company, a leader in the biotechnology industry, is expected to accelerate the development of these treatments as the FDA aims to adapt its regulations to keep pace with scientific advancements. Despite recent stock price gains, CRISpr Therapeutic faces financial challenges, including negative margins and declining revenue growth. Despite these challenges, the company's financial health is supported by strong liquidity metrics and a low debt burden. Despite this, investors should remain cautious about the potential regulatory changes and competitive pressures within the biotech industry.
Published: October 31, 2025
Published by: gurufocus.com
CRISPR Therapeutics Presents New Preclinical Data for CTX460™ De
CRISPR Therapeutics (CRSP) has presented preclinical data for its novel SyNTase™ gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The AATD program, CTX460, is the first investigational candidate to emerge from the SyNTas editing platform and is expected to enter the clinic in mid-2026. The data also indicated specific and durable effects, with >90% mRNA correction, a 5-fold increase in total AAT levels, and >99% serum M-AAT:Z-AATA ratio in AATAD disease models. These results were presented at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress. The company is committed to creating transformative gene-based medicines for serious diseases.
Published: October 10, 2025
Published by: gurufocus.com
CRISPR Therapeutics Highlights Potential Best-in-Class AATD Treatment with CTX460 at ESGCT 2025 Annual Congress
CRISPR Therapeutics (CRSP) has revealed new preclinical data from its SyNTase™ gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The AATD program, CTX460, is the first investigational candidate to emerge from the SyNTas editing platform and is expected to enter the clinic in mid-2026. CTX 460 showed specific and durable effects, with >90% mRNA correction, a 5-fold increase in total AAT levels, and >99% serum M-AAT:Z-AATA ratio in AATAD disease models. The company is focused on creating transformative gene-based medicines for serious diseases. The data were presented at the European Society of Gene and Cell Therapy 2025 Annual Congress.
Published: October 10, 2025
Published by: quiverquant.com
Crispr Therapeutics AG Files $600M Share Offering
On October 15, 2025, CRISPR Therapeutics AG is set to offer and sell additional common shares worth up to $600 million through Jefferies LLC. This move aligns with the company's strategic financial initiatives and could potentially impact its market position and stakeholder interests. The most recent analyst rating on (CRSP) stock is a Buy with a $86.00 price target. The company's overall stock score is primarily influenced by its financial performance, which is challenged by negative profitability and cash flow metrics.
Published: October 15, 2025
Published by: tipranks.com
CRISPR Therapeutics Stock Explodes on Gene-Editing Breakthroughs – Analysts See 35% Upside
CRISPR Therapeutics (NASDAQ: CRSP) shares have surged nearly 40-45% over the past 12 months, with the stock trading at around $69 in mid-October 2025. The company's shares hit a 2025 high near $78 before a modest pullback. Bank of America recently raised its CRSP price target to $93 (from $78), suggesting about 36% upside. The firm plans to raise up to $600 million by selling new shares via an existing agreement with Jefferies, which could dilute existing shares and reflect heavy R&D spending. CRISPR's lead candidate CTX460 achieved >90% gene correction and a 5x increase in serum AAT levels in animal models of alpha-1 antitrypsin deficiency, demonstrating potential for a best-in-class approach to address the disease-causing mutation in >90%. The company plans to begin CTX 460’s first human trial by mid-2026. Pipeline and commercial wins include partner-backed therapy Casgevy for sickle-cell and beta-thalassemia, CTX320 (Lp(a) lowering), CTX310 (targeting ANGPTL3), and CTX340 (hypertension) for cardiovascular and metabolic diseases. Additionally, the company is working on its cardiovascular, metabolic programs.
Published: October 19, 2025
Published by: ts2.tech
FDA to Expedite Gene-Editing Approval Process, Impacting CRSP
The U.S. Food and Drug Administration (FDA) is set to streamline the approval process for personalized gene-editing treatments, potentially accelerating the development of therapies for rare genetic conditions. CRISPR Therapeutics (CRSP) is positioned to benefit from these regulatory changes due to its focus on CRISpr/Cas9-based therapeutics. Despite promising technology, the company faces financial challenges, including negative margins and declining revenue. The company's first approved drug is Casgevy, which targets sickle-cell disease and transfusion-dependent beta-thalassemia. Despite these challenges, analyst sentiment is cautiously optimistic and a target price of $80.72 is expected to rise. However, there are warning signs that the company may be undervalued due to poor business operations.
Published: October 31, 2025
Published by: gurufocus.com
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