Can CRSP's In Vivo Pipeline Aid Long-Term Growth Beyond Casgevy?

CRISPR Therapeutics CRSP is the first and only company in the world to market a CRISPR/Cas9-based therapy. The company tasted major success with the approval of its one-shot gene therapy, Casgevy, 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 company developed Casgevy in partnership with Vertex Pharmaceuticals VRTX.

Per the deal terms, Vertex leads global development, manufacturing and commercialization of Casgevy and shares costs and profits worldwide with CRISPR Therapeutics in a 60:40 ratio.

Following its success with Casgevy, which is an ex vivo therapy, CRISPR Therapeutics is now focusing on in vivo candidates. In contrast to 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 studying its first two in vivo candidates, CTX310 and CTX320, in separate phase I clinical studies, targeting ANGPTL3 and lipoprotein(a), respectively. The company announced updated data from the first 10 patients across the first four cohorts of the phase I study on CTX310 in June. The data showed that a single dose of CTX310 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 86% in LDL.

The data not only highlights the potential of CTX310 but also raises hopes around CRSP’s other in vivo candidate, CTX320, which is being developed in an early-stage for targeting lipoprotein(a), also associated with heart disease.

CRISPR Therapeutics is planning to further expand its in vivo pipeline by advancing two more programs, CTX340 (for refractory hypertension) and CTX450 (for acute hepatic porphyria), into the clinic by the end of 2025.

It can be inferred that CRSP’s efforts to grow its pipeline beyond Casgevy are a big positive for the company, given the potential it holds in the emerging gene therapy market.

However, despite the long-term potential, CRSP’s pipeline is still mostly in the early stages of development. Also, competition for Casgevy from chronic therapies like Bristol Myers’ Reblozyl for TDT and Novartis’ Adakveo for SCD remains a worry.

CRSP’s Competition in the Space

Some companies are also using the CRISPR/Cas9 gene editing technology to address various diseases. One such company is Beam Therapeutics BEAM, which is developing BEAM-101 in the phase I/II BEACON study for treating SCD and TDT. Successful development and a potential approval of BEAM-101 is likely to induce acute competition for CRISPR Therapeutics.

Meanwhile, with regard to in-vivo therapies, Intellia Therapeutics NTLA is one of the few companies with in-vivo CRISPR-based candidates in clinical-stage development.

Intellia is developing multiple in-vivo CRISPR-based therapies in early- to late-stage studies to address different indications like ATTR amyloidosis and hereditary angioedema. Beam Therapeutics is also developing in vivo therapies targeting AATD-associated liver disease and GSDIa indications.

CRSP’s Price Performance, Valuation and Estimates

Shares of CRISPR Therapeutics have rallied 34.7% year to date compared with the industry’s rise of 3.2%.

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 2.68, which is lower than the industry average of 3.10. The stock is trading above its five-year mean of 2.38.

Estimates for CRISPR Therapeutics’ 2025 loss per share have widened from $5.67 to $6.38 in the past 30 days. During the same timeframe, loss per share estimates for 2026 have narrowed from $4.42 to $4.02.

CRSP’s Zacks Rank

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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