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Can CRISPR Build on Casgevy's Success With Its In Vivo Pipeline?
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Key Takeaways
CRSP is advancing CTX310 and CTX320 in early trials targeting heart disease-linked genetic factors.
CTX310 showed up to 82% TG and 81% LDL reduction in early dose-dependent trial results.
CRSP plans to add two more in vivo programs by year-end.
CRISPR Therapeutics (CRSP - Free Report) is currently the only company in the world to have secured approval for a CRISPR-based gene therapy developed in partnership with Vertex Pharmaceuticals. Following the landmark success with Casgevy, an ex vivo therapy, the company is now focusing on in vivo candidates. Unlike ex vivo therapies, where cells are removed, modified and then inserted back into one’s body, in vivo therapies involve infusing new genes directly into the body.
CRISPR Therapeutics is currently evaluating two in vivo candidates, namely CTX310 and CTX320, in separate early-stage studies, targeting ANGPTL3 and lipoprotein(a) [Lp(a)], respectively — both associated with atherosclerotic heart disease.
Last month, CRSP reported initial top-line data from the first 10 patients across the first four cohorts of the phase I study on CTX310. A single dose demonstrated dose-dependent decreases in low-density lipoprotein (LDL) and triglyceride (TG) levels, with peak reduction of up to 82% in TG and up to 81% in LDL.
These results have raised excitement around CTX320, a readout from which is expected later in the second quarter of 2025. CRISPR Therapeutics also intends to advance two additional in vivo programs into the clinic by year-end, further expanding its footprint in this emerging modality.
CRSP’s Competition in this Space
The company is currently lagging behind Intellia Therapeutics (NTLA - Free Report) , which is one of the few companies with multiple clinical-stage in vivo gene editing programs. Intellia is advancing two candidates — NTLA-2002 for hereditary angioedema (HAE) and nex-z (NTLA-2001) for transthyretin (ATTR) amyloidosis — across late-stage development. Intellia Therapeutics plans to submit a regulatory filing with the FDA for the HAE therapy next year, making it a front-runner in the space.
Beam Therapeutics (BEAM - Free Report) is also actively developing in vivo candidates. Beam Therapeutics is currently conducting separate phase I/II studies for BEAM-302 targeting alpha-1 antitrypsin deficiency (AATD) and BEAM-301 for glycogen storage disease 1a (GSD1a). While earlier in development compared to Intellia Therapeutics, Beam Therapeutics' base-editing approach offers a differentiated pathway that could complement broader in vivo innovation.
CRSP’s Price Performance, Valuation and Estimates
Shares of CRISPR Therapeutics have underperformed the industry year to date, as seen in the chart below.
Image Source: Zacks Investment Research
From a valuation standpoint, CRSP is trading at a discount to the industry. Going by the price-to-book value (P/B) ratio, the company’s shares currently trade at 1.80, lower than 3.10 for the industry. The stock is also trading below its five-year mean of 2.41.
Image Source: Zacks Investment Research
Estimates for CRISPR’s 2025 loss per share have widened from $4.96 to $5.54 in the past 60 days. During the same timeframe, loss per share estimates for 2026 have increased from $3.65 to $4.11.
Image: Bigstock
Can CRISPR Build on Casgevy's Success With Its In Vivo Pipeline?
Key Takeaways
CRISPR Therapeutics (CRSP - Free Report) is currently the only company in the world to have secured approval for a CRISPR-based gene therapy developed in partnership with Vertex Pharmaceuticals. Following the landmark success with Casgevy, an ex vivo therapy, the company is now focusing on in vivo candidates. Unlike ex vivo therapies, where cells are removed, modified and then inserted back into one’s body, in vivo therapies involve infusing new genes directly into the body.
CRISPR Therapeutics is currently evaluating two in vivo candidates, namely CTX310 and CTX320, in separate early-stage studies, targeting ANGPTL3 and lipoprotein(a) [Lp(a)], respectively — both associated with atherosclerotic heart disease.
Last month, CRSP reported initial top-line data from the first 10 patients across the first four cohorts of the phase I study on CTX310. A single dose demonstrated dose-dependent decreases in low-density lipoprotein (LDL) and triglyceride (TG) levels, with peak reduction of up to 82% in TG and up to 81% in LDL.
These results have raised excitement around CTX320, a readout from which is expected later in the second quarter of 2025. CRISPR Therapeutics also intends to advance two additional in vivo programs into the clinic by year-end, further expanding its footprint in this emerging modality.
CRSP’s Competition in this Space
The company is currently lagging behind Intellia Therapeutics (NTLA - Free Report) , which is one of the few companies with multiple clinical-stage in vivo gene editing programs. Intellia is advancing two candidates — NTLA-2002 for hereditary angioedema (HAE) and nex-z (NTLA-2001) for transthyretin (ATTR) amyloidosis — across late-stage development. Intellia Therapeutics plans to submit a regulatory filing with the FDA for the HAE therapy next year, making it a front-runner in the space.
Beam Therapeutics (BEAM - Free Report) is also actively developing in vivo candidates. Beam Therapeutics is currently conducting separate phase I/II studies for BEAM-302 targeting alpha-1 antitrypsin deficiency (AATD) and BEAM-301 for glycogen storage disease 1a (GSD1a). While earlier in development compared to Intellia Therapeutics, Beam Therapeutics' base-editing approach offers a differentiated pathway that could complement broader in vivo innovation.
CRSP’s Price Performance, Valuation and Estimates
Shares of CRISPR Therapeutics have underperformed the industry year to date, as seen in the chart below.
From a valuation standpoint, CRSP is trading at a discount to the industry. Going by the price-to-book value (P/B) ratio, the company’s shares currently trade at 1.80, lower than 3.10 for the industry. The stock is also trading below its five-year mean of 2.41.
Estimates for CRISPR’s 2025 loss per share have widened from $4.96 to $5.54 in the past 60 days. During the same timeframe, loss per share estimates for 2026 have increased from $3.65 to $4.11.
CRISPR Therapeutics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.