Can Casgevy Deliver a Turnaround for CRISPR Therapeutics?

CRISPR Therapeutics’ CRSP first marketed product is the one-shot gene therapy Casgevy, approved in late 2023 and early 2024 across the United States and Europe for two blood disorder indications — sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The therapy was developed in partnership with Vertex Pharmaceuticals VRTX, which leads global development and commercialization. While VRTX is responsible for recording all product sales, CRSP records its share of Casgevy’s profits/losses.

While Casgevy is the first-ever approved CRISPR/Cas9 gene-edited therapy, its initial commercial uptake has been modest. Despite the groundbreaking nature of the therapy, Vertex recorded just $10 million in product revenues for full-year 2024, highlighting the complexity of the treatment process. Patients must first undergo stem cell collection, followed by ex vivo gene editing and reinfusion, making it labor-intensive and time-consuming.

Despite these hurdles, Casgevy offers the potential to be a one-time functional cure for SCD and TDT. The eligible patient population for both indications is estimated to be approximately 60,000 across approved markets. To support broader access, Vertex is ramping up its commercial efforts. As of May 1, more than 65 authorized treatment centers (ATCs) had been activated globally, and nearly 90 patients had their first cell collection. New patient starts are expected to grow significantly throughout 2025. Our model estimates Vertex to record Casgevy sales of about $99 million this year.

CRSP’s Competition for Casgevy

While Casgevy is currently the only CRISPR-based therapy, several companies are advancing gene-editing treatments using similar technology. Beam Therapeutics BEAM is developing its lead candidate, BEAM-101, for SCD. Earlier this month, Beam Therapeutics reported updated data from a phase I/II study, which showed that treatment with BEAM-101 led to a robust and durable increase in fetal hemoglobin and a reduction in sickle hemoglobin.

Casgevy also competes with already-approved therapies like Bristol Myers’ BMY Reblozyl for TDT and Novartis’ NVS Adakveo for SCD. Unlike Casgevy, which is marketed at an extremely high price of $2.2 million, the Bristol Myers and Novartis drugs are available at a fraction of Casgevy’s cost, making them more accessible to many patients despite their chronic use.

CRSP’s Price Performance, Valuation and Estimates

Shares of CRISPR Therapeutics have outperformed the industry year to date, as seen in the chart below.

From a valuation standpoint, CRSP is trading at a discount to the industry. Based on the price-to-book value (P/B) ratio, the company’s shares currently trade at 2.10, which is lower than the industry average of 3.04. The stock is also trading below its five-year mean of 2.39.

Estimates for CRISPR’s 2025 loss per share have widened from $5.06 to $5.54 in the past 60 days. During the same timeframe, loss per share estimates for 2026 have increased from $3.76 to $4.30.

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.

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This article originally published on Zacks Investment Research (zacks.com).

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