S&P 500 could hit 8,000 in 2026 on more easing from Fed: JPMorgan
CRISPR Therapeutics stands at a critical juncture as its gene editing technology transitions from clinical promise to commercial reality. The company, focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform, has made significant strides in its pipeline development while navigating the challenges of bringing novel therapies to market.
Pipeline Progress Drives Future Value
The third quarter of 2025 has been marked by substantial progress across CRISPR’s development programs. The company’s lead product, Casgevy for sickle cell disease (SCD) and thalassemia (TDT), continues its steady launch trajectory. While initial revenue recognition has been slower than some anticipated, management has indicated strong payer coverage and strategic authorized treatment center (ATC) footprint expansion.
CRISPR’s in-vivo editing pipeline has emerged as a particular area of interest for investors and pharmaceutical companies alike. CTX310, which targets ANGPTL3 for dyslipidemia, has shown promising Phase 1 data with dose-dependent decreases in ANGPTL3 and significant reductions in triglycerides and LDL cholesterol. This data validates the company’s liver-targeting capabilities and positions CTX310 as a potential best-in-class therapy.
The anticipated data from CTX320, targeting Lp(a), has been delayed to the second half of 2026. This delay may be connected to Novartis’s postponement of their Phase 3 LPA-targeting siRNA pelacarsen data. Complete Phase 1 data for CTX310 is expected to be presented at a medical meeting in the second half of 2025.
In the immuno-oncology and autoimmune space, CRISPR is advancing CTX112 and CTX131. CTX112 is positioned to potentially offer similar efficacy to autologous CAR-T therapies but with improved safety, convenience, and pricing around $100,000, making it commercially viable.
Strategic Partnerships Expand Therapeutic Reach
In May 2025, CRISPR announced a strategic collaboration with Sirius Therapeutics to develop SRSD107, a long-acting Factor XI small interfering RNA (siRNA) for thromboembolic disorders. The deal involved an upfront payment of $25 million plus a $70 million equity investment in Sirius for co-development and co-commercialization rights.
This partnership represents a strategic diversification beyond gene editing into siRNA-based approaches, enhancing CRISPR’s cardiovascular franchise. SRSD107 is advancing into Phase 2 clinical trials and could be pivotal trial ready next year, targeting significant opportunities in thromboembolic disorders with potential applications in various bleeding and cardiovascular-related indications.
The siRNA approach may offer advantages over daily oral anticoagulants and IV antibodies due to its potential six-month dosing schedule and faster response management for breakthrough bleeds or surgery. This collaboration could lead to a best-in-class treatment option with bi-annual dosing, providing greater convenience than current treatments.
Financial Position Supports Long-term Strategy
CRISPR maintains a robust financial foundation with approximately $1.86 billion in cash as of Q1 2025. This strong cash position provides runway for continued pipeline development and strategic initiatives. However, the company continues to operate at a loss, with negative EPS forecasts of -6.52 for FY1 and -6.91 for FY2.
The market capitalization has fluctuated between $2.86 billion and $5.20 billion during 2025, reflecting both the volatility in biotech stocks and changing investor sentiment around CRISPR’s prospects. The slow initial launch of Casgevy has tempered near-term revenue expectations, with 2025 sales now modeled at $114 million according to some estimates.
Market Positioning and Competitive Landscape
CRISPR Therapeutics has established itself as a leader in the gene editing space, with Casgevy representing the first approved CRISPR-based therapy. The company has outperformed the XBI biotech index year-to-date, suggesting investor confidence in its long-term prospects despite near-term challenges.
The company’s expansion into siRNA therapies through the Sirius partnership demonstrates a strategic broadening beyond its core gene editing technology. This diversification reduces single modality risk and opens new therapeutic avenues.
Competition remains robust across CRISPR’s target indications. In the cardiovascular space, companies like Novartis are advancing competing therapies. The gene therapy landscape continues to evolve rapidly, with regulatory and reimbursement frameworks still developing.
Bear Case
How concerning are the negative EPS projections?
CRISPR’s projected negative earnings per share of -6.52 for FY1 and -6.91 for FY2 represent a significant financial challenge. These losses reflect the substantial ongoing investment required to advance multiple clinical programs simultaneously while building commercial infrastructure for Casgevy.
The biotechnology sector is accustomed to extended periods of negative earnings during development phases. However, the depth of these projected losses raises questions about the timeline to profitability. Without substantial revenue acceleration from Casgevy or partnership deals, CRISPR may need to consider additional financing options in the future, potentially diluting existing shareholders.
The slower-than-expected revenue recognition from Casgevy compounds these concerns. Initial sales projections have been revised downward, with 2025 revenue estimates reduced to $10.1 million from a previous $47.2 million according to some analysts. This adjustment reflects the complex reimbursement landscape and logistical challenges of cell therapy administration.
What risks does the delayed CTX320 data present?
The postponement of CTX320 data to the second half of 2026 removes a near-term catalyst that investors had anticipated. This delay may cause concern among those looking for validation of CRISPR’s in-vivo editing platform across multiple targets.
The timing coincides with Novartis’s delay in their Phase 3 LPA-targeting siRNA pelacarsen data, suggesting potential broader challenges in this therapeutic approach. When CTX320 data eventually emerges, it will face direct comparison with Novartis’s results, creating a binary risk event for CRISPR.
This delay also impacts the company’s ability to advance CTX320 into later-stage trials, potentially extending the timeline to market and allowing competitors to gain ground. For a company still establishing commercial validation of its technology platform, such delays can affect investor confidence and strategic partnership opportunities.
Bull Case
How might the promising CTX310 data impact CRISPR’s valuation?
The positive Phase 1 data from CTX310 provides crucial validation for CRISPR’s in-vivo liver editing capabilities. The dose-dependent decreases in ANGPTL3 and significant reductions in triglycerides and LDL cholesterol position CTX310 as a potentially best-in-class therapy for dyslipidemia.
This data strengthens CRISPR’s position in the cardiovascular space, a massive market opportunity with billions in potential revenue. The ability to demonstrate meaningful lipid reduction with good safety profiles suggests CTX310 could address unmet needs in conditions like Homozygous Familial Hypercholesterolemia (HoFH), severe Hypertriglyceridemia (sHTG), Heterozygous Familial Hypercholesterolemia (HeFH), and Metabolic Dyslipidemia (MDL).
Success with CTX310 would validate CRISPR’s platform beyond rare diseases like sickle cell disease, demonstrating applicability to more common conditions with larger patient populations. This expansion of addressable markets could dramatically increase the company’s valuation multiple as investors recognize the broader commercial potential of the technology platform.
What potential does the Sirius Therapeutics partnership offer?
The collaboration with Sirius Therapeutics represents a strategic diversification beyond CRISPR’s core gene editing technology. By expanding into siRNA therapeutics, CRISPR reduces its reliance on a single technological approach and broadens its therapeutic reach.
SRSD107, the lead asset from this partnership, addresses thromboembolic disorders—a substantial market opportunity with applications across various cardiovascular and bleeding-related indications. The potential for bi-annual dosing offers a significant advantage over daily oral anticoagulants, potentially improving patient compliance and outcomes.
The deal structure, with $25 million upfront plus a $70 million equity investment, provides CRISPR with co-development and co-commercialization rights. This 50-50 cost and profit-sharing model could deliver substantial returns if SRSD107 succeeds in clinical trials and reaches commercialization. The partnership also includes access to two future siRNA programs, further expanding CRISPR’s pipeline potential.
SWOT Analysis
Strengths
Validated gene editing technology with approved therapy Casgevy for SCD/TDT
Diversified pipeline across multiple therapeutic areas
Strong cash position of approximately $1.86 billion
Strategic partnerships expanding technological capabilities
Promising early data from in-vivo editing programs
Experienced management team with expertise in genetic medicine
Weaknesses
Negative earnings projections for foreseeable future
Slower than expected Casgevy launch and revenue recognition
Delayed data readouts for some key programs
High cash burn rate supporting multiple clinical programs
Reliance on complex manufacturing processes for cell therapies
Opportunities
Multiple data readouts throughout 2025 could drive valuation
Expansion into common diseases with larger market potential
Growing pharmaceutical interest in gene editing technologies
Potential for additional strategic partnerships
Accelerating Casgevy uptake with expanding reimbursement coverage
Diversification beyond gene editing into complementary modalities
Threats
Regulatory uncertainties for novel genetic medicines
Competitive landscape with multiple companies pursuing similar targets
Clinical trial risks inherent to novel therapeutic approaches
Potential intellectual property challenges
Pricing pressures from payers for high-cost genetic therapies
Manufacturing and scalability challenges for cell and gene therapies
Analysts Targets
Barclays - November 11, 2025 - Equal Weight - $56.00
JMP Securities - June 27, 2025 - Market Outperform - $86.00
JMP Securities - May 21, 2025 - Market Outperform - $86.00
Piper Sandler - May 20, 2025 - Overweight - $105.00
H.C. Wainwright & Co. - May 20, 2025 - Buy - $65.00
Barclays - May 9, 2025 - Equal Weight - $42.00
Clear Street - May 7, 2025 - Buy - $45.00
JMP Securities - May 7, 2025 - Market Outperform - $86.00
BMO Capital Markets - April 22, 2025 - Outperform - $97.00
This analysis is based on information available through November 11, 2025.
InvestingPro: Smarter Decisions, Better Returns
Gain an edge in your investment decisions with InvestingPro’s in-depth analysis and exclusive insights on CRSP. Our Pro platform offers fair value estimates, performance predictions, and risk assessments, along with additional tips and expert analysis. Explore CRSP’s full potential at InvestingPro.
Should you invest in CRSP right now? Consider this first:
Investing.com’s ProPicks, an AI-driven service trusted by over 130,000 paying members globally, provides easy-to-follow model portfolios designed for wealth accumulation. Curious if CRSP is one of these AI-selected gems? Check out our ProPicks platform to find out and take your investment strategy to the next level.
To evaluate CRSP further, use InvestingPro’s Fair Value tool for a comprehensive valuation based on various factors. You can also see if CRSP appears on our undervalued or overvalued stock lists.
These tools provide a clearer picture of investment opportunities, enabling more informed decisions about where to allocate your funds.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.