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CRISPR Therapeutics (CRSP) Is Up 7.9% After Unveiling CTX460 Gene Editing Data for AAT Deficiency Therapy – Has The Bull Case Changed?

Reviewed by Sasha Jovanovic
- At the recent European Society of Gene and Cell Therapy Congress, CRISPR Therapeutics presented new preclinical data from its SyNTase gene editing platform showing that CTX460 achieved over 90% mRNA correction and a five-fold increase in total AAT levels in animal models of Alpha-1 Antitrypsin Deficiency.
- This marks the first public data for the SyNTase platform, positioning CRISPR Therapeutics as an innovator in addressing the root genetic cause of this rare disease through a single-dose therapy.
- We will explore how the strong preclinical results for CTX460, especially its precise gene correction capabilities, influence the investment narrative for CRISPR Therapeutics.
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What Is CRISPR Therapeutics' Investment Narrative?
CRISPR Therapeutics continues to attract attention for its leadership in gene editing, but being a shareholder still comes down to a belief in the company's ability to transition from preclinical innovation to commercial success. The recent SyNTase/CTX460 data is compelling and broadens the narrative around CRISPR's technical edge, underscoring its push to address previously untreatable diseases. For now, this news elevates the potential of the pipeline but doesn't materially alter the most important short term catalysts, which remain centered on clinical trial milestones and commercial uptake of approved therapies like CASGEVY. The risks, however, are shifting slightly, greater visibility from these breakthroughs can heighten expectations, but CRISPR remains unprofitable with losses widening and revenue growth still modest compared to future projections. This means dilution, competition, and clinical trial outcomes stay front of mind for investors following the stock closely. On the other hand, the company’s expanding losses are a risk investors should be aware of.
CRISPR Therapeutics' shares have been on the rise but are still potentially undervalued by 45%. Find out what it's worth.Exploring Other Perspectives
Explore 20 other fair value estimates on CRISPR Therapeutics - why the stock might be worth less than half the current price!
Build Your Own CRISPR Therapeutics Narrative
Disagree with this assessment? Create your own narrative in under 3 minutes - extraordinary investment returns rarely come from following the herd.
- A great starting point for your CRISPR Therapeutics research is our analysis highlighting 2 key rewards and 1 important warning sign that could impact your investment decision.
- Our free CRISPR Therapeutics research report provides a comprehensive fundamental analysis summarized in a single visual - the Snowflake - making it easy to evaluate CRISPR Therapeutics' overall financial health at a glance.
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This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.
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About NasdaqGM:CRSP
CRISPR Therapeutics
A gene editing company, focuses on developing gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform.
Excellent balance sheet and fair value.
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