Is CRISPR Therapeutics Still a Viable Long-Term Play Despite a Delayed Revenue Ramp?

CRISPR Therapeutics (NASDAQ: CRSP) has long been a focal point for investors seeking exposure to the transformative potential of gene-editing technologies. However, with its revenue ramp lagging expectations and a net loss of $106.4 million in Q3 2025, skepticism about its long-term viability persists. This analysis evaluates whether the company's evolving pipeline-centered on zugo-cel and Casgevy-justifies its current valuation and whether patient investors should consider it a compelling long-term opportunity.

Clinical Progress: Zugo-cel and Casgevy as Pillars of Potential

CRISPR's allogeneic CAR T therapy, zugo-cel, has demonstrated robust clinical progress in 2025, particularly in autoimmune diseases and hematologic malignancies. In trials for systemic lupus erythematosus (SLE) and immune-mediated necrotizing myopathy, four patients treated with a 100 million cell dose achieved sustained B-cell depletion for at least 28 days, with one SLE patient entering DORIS remission through Month 6. For relapsed/refractory large B-cell lymphoma (LBCL), zugo-cel delivered an overall response rate of 90% and a complete response rate of 70% at the recommended Phase 2 dose of 600 million cells, with 67% of responders maintaining remission after 12 months. These results position zugo-cel as a promising candidate for both autoimmune and oncology indications, supported by a favorable safety profile, including no high-grade cytokine release syndrome (CRS) at lower doses as reported in the company's Q3 2025 update.

Meanwhile, Casgevy, CRISPR's first approved CRISPR-based therapy for sickle cell disease and transfusion-dependent beta-thalassemia, has shown strong commercial traction. As of September 30, 2025, 39 patients had received infusions, with Vertex Pharmaceuticals projecting 2025 revenue exceeding $100 million. The therapy's pediatric development is also advancing, with Phase 3 trials in children with sickle cell disease and beta-thalassemia completed, and data slated for presentation at the American Society of Hematology (ASH) meeting in December 2025. Additionally, CRISPR's in vivo gene-editing platform, exemplified by CTX310™, has shown dose-dependent reductions of up to 82% in triglycerides and 86% in LDL cholesterol in Phase 1 trials, highlighting its potential in metabolic diseases.

Financials and Valuation: A Tale of Promise and Risk

Despite its clinical advancements, CRISPR's financials remain a concern. The company reported a net loss of $106.4 million in Q3 2025 but maintains a strong cash position of $1.94 billion, providing a runway to advance its pipeline. Analysts argue the stock is undervalued, with a discounted cash flow (DCF) model estimating an intrinsic value of $126.91 per share and Morningstar assigning a fair value of $106 per share-both significantly above the current price of approximately $57.91. However, the company's price-to-book ratio of 2.7x, while below the peer group average of 20.2x, reflects conservative investor expectations given the high execution risks associated with commercializing first-in-class therapies.

CRISPR's valuation of $5.4 billion in 2025 appears justified by its first-mover advantage in the CRISPR market. Its partnership with Vertex for Casgevy provides access to a global commercial infrastructure, while its cash reserves outpace those of competitors like Editas Medicine and Intellia Therapeutics according to market analysis. In contrast, Editas remains in clinical-stage development, and Intellia has faced setbacks, including a clinical hold on one of its therapies due to a patient's death as reported by market observers. The global CRISPR market, projected to grow at a 15.3% CAGR through 2035, further supports CRISPR's long-term potential.

Competitive Landscape: Strengths and Challenges

CRISPR's competitive positioning is bolstered by its diversified pipeline and commercialized product. While Editas and Intellia focus on in vivo gene editing, CRISPR's allogeneic CAR T therapies and partnerships with industry leaders like Lilly for zugo-cel in combination with pirtobrutinib expand its therapeutic reach. Additionally, its SyNTase™ platform and CTX460™ preclinical data underscore its innovation in in vivo gene correction as highlighted in the Q3 2025 business update. However, challenges remain, including regulatory scrutiny for new therapies and the high costs of scaling gene-editing manufacturing.

Conclusion: A High-Risk, High-Reward Proposition

CRISPR Therapeutics' long-term viability hinges on its ability to translate clinical success into commercial revenue. While Casgevy's approval and zugo-cel's promising data justify optimism, the company's delayed revenue ramp and high cash burn necessitate a patient, risk-tolerant investor. Analysts' valuation estimates and the growing CRISPR market suggest the stock is undervalued, but execution risks-such as regulatory delays or safety concerns-could hinder progress. For investors with a multi-year horizon and a tolerance for volatility, CRISPR remains a compelling play on the future of gene editing, provided its pipeline continues to deliver.