CRISPR Therapeutics CRSP stock rose more than 25% in the past week, thanks to the encouraging fourth-quarter 2024 results and a rating upgrade by a Wall Street analyst.

Though CRSP’s quarterly earnings and sales declined year over year, they significantly beat estimates. In the year-ago period, CRISPR Therapeutics received a major milestone payment from partner Vertex Pharmaceuticals VRTX in connection with the approval of one-shot gene therapy Casgevy for two blood disorders.

Alongside earnings results, management also cited pipeline advancements and updates on nearly all its pipeline candidates expected throughout this year. Based on the positive expectations for the company’s clinical programs, an analyst at Evercore ISI upgraded CRSP’s rating from "In Line" to "Outperform" while also significantly raising the target price by 65% to $99.

Let’s delve into the company’s strengths and weaknesses to gain a better understanding of how to play the stock amid this price rise.

CRSP/VRTX’s Strong Global Launch Efforts for Casgevy

The approval for Casgevy is a breakthrough for medical science, as it is the first approval for a CRISPR-based gene-editing therapy in the world. Developed in collaboration with Vertex Pharmaceuticals, Casgevy was approved in late 2023/early 2024 for two indications — sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) — in the United States and Europe.

Both SCD and TDT indications have a significant unmet medical need. With Casgevy’s approval, CRISPR Therapeutics offers a new treatment option to an estimated 35,000 patients living with severe SCD or TDT across the United States and Europe, with additional patients in Bahrain and Saudi Arabia. The therapy has demonstrated the potential to alleviate blood transfusion requirements for TDT patients as well as reduce painful and debilitating sickle crises for SCD patients.

Per management, more than 50 authorized treatment centers (ATCs) were activated globally as of the end of 2024 and more than 50 patients had at least one cell collection across all regions. New patient starts are expected to grow significantly throughout 2025. Vertex, in its fourth-quarter earnings release reported last week, recorded $8 million in product revenues from Casgevy sales.

CRSP’s Pipeline Programs Show Promise

CRISPR Therapeutics is pursuing the development of CRISPR candidates to create novel CAR-T cell therapies. It is developing two next-generation CAR-T therapy candidates — CTX112 (targeting CD19-positive B-cell malignancies) and CTX131 (targeting relapsed or refractory solid tumors) — in separate phase I/II studies.

In December 2024, CRSP presented positive preliminary results from the CTX112 study at a major medical meeting. These results showed strong efficacy comparable to autologous therapies, a tolerable safety profile and robust cell expansion. Management intends to discuss these results with regulatory authorities to align on the path forward for CTX112 in B-cell malignancies. An update on the same is expected in mid-2025.

CRSP is also exploring the potential of these next-generation candidates in other indications. Last year, management started two new clinical studies — one evaluating CTX131 for hematological malignancies (including T-cell lymphomas) and another assessing CTX112 in systemic lupus erythematosus indication, systemic sclerosis and inflammatory myositis. Updates from both these studies are expected later this year.

Following the success with ex-vivo therapy Casgevy, management is 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. CRSP is currently evaluating two in-vivo candidates, namely CTX310 (targeting ANGPTL3) and CTX320 (targeting lipoprotein(a) [Lp(a)]), in separate early-stage studies. These are directed toward validated therapeutic targets associated with cardiovascular disease. Management intends to further expand this in-vivo pipeline with two new in-vivo programs before 2025-end.

Stiff Competition for CRSP’s Targeted Markets

Some other companies are also using CRISPR technology to address various ailments. One such company is Beam Therapeutics BEAM, which is pursuing an ex vivo, autologous transplant-based approach for SCD and TDT indications.

BEAM is developing its lead pipeline candidate BEAM-101 in SCD indication in the phase I/II BEACON study. The company is also advancing its engineered stem cell antibody paired evasion (ESCAPE) conditioning strategy to face the toxicity challenges of autologous transplant in SCD and beta-thalassemia patients.

With regard to in-vivo therapies, CRSP is currently lagging Intellia Therapeutics NTLA, which is one of the few companies with in-vivo CRISPR-based candidates in clinical-stage development. NTLA is developing multiple in-vivo CRISPR-based therapies in early to late-stage studies to address different indications like ATTR amyloidosis and hereditary angioedema.

CRSP Stock’s Performance, Valuation & Estimates

Shares of CRISPR Therapeutics have plunged nearly 40% in the past year compared with the industry’s 9% decline, as seen in the chart below. The stock has also underperformed the sector and the S&P 500 index. However, shares of the company are currently trading above its 50-day and 200-day moving averages.

CRSP Stock Underperforms Industry, Sector & S&P 500

Image Source: Zacks Investment Research

From a valuation standpoint, CRISPR Therapeutics 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 2.20, lower than 3.22 for the industry.

Image Source: Zacks Investment Research

Estimates for CRISPR’s 2025 loss per share have improved from $5.30 to $5.07 in the past seven days. During the same timeframe, loss per share estimates for 2026 have widened from $2.69 to $4.10.

Image Source: Zacks Investment Research

How to Play CRSP Stock?

We would suggest holding on to this Zacks Rank #3 (Hold) stock for now, considering its growth prospects. While we acknowledge that most of CRISPR’s pipeline is still in early-stage development, having a marketed product like Casgevy ensures that it has now overcome its biggest hurdle — the lack of a stable revenue stream.

With Casgevy sales picking up pace, the company’s strong cash balance of around $1.9 billion (as of 2024-end) should also comfort investors that it can smoothly carry out its day-to-day operations, including late-stage development of its pipeline. Unlike last year, when CRSP did not provide any major update on its pipeline, it has assured investors of updates throughout this year, which could serve as a catalyst for the stock.

You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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