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ZUG, Switzerland and BOSTON - CRISPR Therapeutics (NASDAQ:CRSP), a $6.8 billion gene editing company whose stock has surged over 90% year-to-date, presented new preclinical data for its alpha-1 antitrypsin deficiency (AATD) treatment candidate at the European Society of Gene and Cell Therapy Annual Congress, according to a company press release. InvestingPro data shows the company trading near its 52-week high of $78.48, reflecting strong investor confidence in its therapeutic pipeline.
The investigational therapy, CTX460, is the first candidate from CRISPR’s SyNTase gene editing platform and demonstrated significant efficacy in AATD disease models, achieving over 90% mRNA correction and more than 99% serum M-AAT:Z-AAT ratio following a single dose.
CTX460 uses lipid nanoparticle delivery to target the E342K mutation in the SERPINA1 gene that causes AATD, a genetic disorder where patients produce a misfolded version of the alpha-1 antitrypsin protein. This misfolding leads to protein accumulation in the liver and insufficient protection for lung tissue.
In preclinical studies using both mouse and rat AATD models, a single dose of CTX460 achieved significant gene correction at doses as low as 0.1 mg/kg. At a 0.5 mg/kg dose, the therapy produced a more than five-fold increase in total serum AAT levels compared to pre-treatment values.
"The preclinical results demonstrate the potential of CTX460, developed using our novel SyNTase editing platform, to correct the mutation with precision and efficiency," said Naimish Patel, Chief Medical Officer of CRISPR Therapeutics.
The company expects to initiate clinical trials for CTX460 in mid-2026. Current AATD treatments require weekly intravenous infusions of purified protein and do not address the underlying genetic cause of the disease.
AATD affects the liver and lungs, with patients typically having alpha-1 antitrypsin levels approximately four times lower than normal, leaving them vulnerable to emphysema and other serious conditions.
The durability of editing was maintained for up to 9 weeks in mice and 7 weeks in rats, according to the data presented at the conference. While the company maintains a strong financial health score according to InvestingPro analysis, with a current ratio of 16.6 indicating robust liquidity, investors should note that analysts expect sales to decline in the current year. The company is scheduled to report its next earnings on October 29, which could provide further insights into CTX460’s development timeline. For comprehensive analysis of CRISPR Therapeutics’ financial health and growth prospects, including 15+ additional ProTips, investors can access the detailed Pro Research Report available on InvestingPro.
In other recent news, CRISPR Therapeutics has reported significant developments that could interest investors. The company’s second-quarter 2025 financial results highlighted CASGEVY sales reaching $30 million, marking a 114% increase from the first quarter’s $14 million. CRISPR Therapeutics, in collaboration with Sirius Therapeutics, has also dosed the first patient in a Phase 2 clinical trial for SRSD107, a therapy aimed at preventing venous thromboembolism in knee surgery patients. This trial is part of a broader partnership with Sirius that has sparked interest among analysts.
BMO Capital has reiterated its Outperform rating with a $75 price target, citing potential in the thrombosis market. Meanwhile, H.C. Wainwright has maintained a Buy rating and increased its price target to $80, noting that the current target excludes potential contributions from SRSD107. JPMorgan has initiated coverage with an Overweight rating and a $70 price target, suggesting that CRISPR Therapeutics is approaching several critical points in its portfolio. These updates reflect analysts’ positive outlooks on CRISPR Therapeutics’ strategic moves and market potential.
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