Intellia Halts Major Trial Following Severe Liver Incident, Shares Plummet
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Oct 27 2025
0mins
Should l Buy CRSP?
Source: Benzinga
Trial Pause: Intellia Therapeutics has paused patient dosing and screening in its Phase 3 MAGNITUDE trials for its CRISPR therapy, nexiguran ziclumeran (nex-z), due to a serious liver-related adverse event in a participant.
Patient Safety Commitment: The company is working with regulators and medical experts to assess the incident, emphasizing its commitment to patient safety and planning to safely resume enrollment.
Promising Phase 1 Results: Long-term follow-up data from a Phase 1 trial showed significant reductions in transthyretin protein levels and improvements in neuropathy scores among patients treated with nex-z.
Market Impact: Following the announcement of the trial pause, Intellia's shares fell by over 43%, reflecting investor concerns about the implications for the ongoing MAGNITUDE trials.
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Analyst Views on CRSP
Wall Street analysts forecast CRSP stock price to rise
10 Analyst Rating
8 Buy
2 Hold
0 Sell
Strong Buy
Current: 60.140
Low
50.00
Averages
78.89
High
105.00
Current: 60.140
Low
50.00
Averages
78.89
High
105.00
About CRSP
CRISPR Therapeutics AG is a Switzerland-based gene editing company focused on the development of CRISPR/Cas9-based therapeutics. CRISPR/Cas9 stands for Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) and is a technology for gene editing, the process of precisely altering specific sequences of genomic DNA. The Company aims to apply this technology to disrupt, delete, correct and insert genes to treat genetically-defined diseases and to engineer advanced cellular therapies. The Company has acquired the rights to the intellectual property (IP) encompassing CRISPR/Cas9 and related technologies and is also involved in its own IP research and additional in-licensing efforts. The Company product development and partnership strategies are designed to exploit the full potential of the CRISPR/Cas9 platform while maximizing the probability of successfully developing their product candidates.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
CRISPR Therapeutics: Potential and Challenges in Gene Editing
- Therapeutic Progress: CRISPR's gene-editing therapy Casgevy was approved in 2023 for treating sickle cell disease and transfusion-dependent beta thalassemia, showcasing its revolutionary potential in healthcare.
- High Treatment Costs: Despite Casgevy's staggering price of $2.2 million, healthcare experts justify this cost due to its one-time treatment nature, which could save patients time and money in the long run.
- Financial Challenges: CRISPR reported a net loss of $581.6 million in 2025, an increase from the $366.3 million loss in 2024, highlighting the significant expenditure pressures the company faces as it rolls out Casgevy.
- Strong Cash Position: As of the end of last year, CRISPR had approximately $2 billion in cash and marketable securities, indicating that despite its losses, the company is not at immediate risk of running out of funds, maintaining long-term growth potential.
See More
CRISPR Therapeutics Faces Significant Challenges Amid Slow Rollout
- Escalating Financial Losses: CRISPR reported a net loss exceeding $580 million in 2025, a significant increase from the $366.3 million loss in 2024, highlighting the pressure from high expenditures during the rollout of the Casgevy treatment and ongoing financial struggles.
- Strong Cash Position: Despite the losses, CRISPR maintained approximately $2 billion in cash and marketable securities at the end of last year, which is sufficient to support its daily operations, having consumed $345 million in 2025, indicating that the company is not at immediate risk of running out of funds.
- Slow Product Rollout: Although Casgevy was approved in 2023 and patient initiations nearly tripled last year, its staggering price of $2.2 million and profit-sharing with Vertex Pharmaceuticals limit CRISPR's market earnings, adversely affecting its profitability.
- Uncertain Market Outlook: While CRISPR's gene-editing therapies hold long-term potential, the lack of current profitability and the risk assessment of its stock make it a cautious buy for investors, especially as it was not included in the list of top recommended stocks by analysts.
See More
Cathie Wood's Latest Investment Moves in Ark Innovation ETF
- Figma Investment Update: Ark Innovation ETF purchased additional shares of Figma on February 23, making it the 38th largest holding at 0.66% of the total portfolio; despite concerns about slowing growth, Figma's 40% year-over-year sales increase in Q4 indicates strong market potential.
- CRISPR Holdings Overview: On February 20, Ark increased its stake in CRISPR Therapeutics, now the second-largest holding at 6.64% of the fund, with only $3.5 million in sales last year, yet the potential commercialization of gene-editing drugs could lead to explosive growth, representing a high-risk, high-reward investment.
- CoreWeave Investment Outlook: Ark also bought more shares of CoreWeave on February 20, making it the 18th largest holding at approximately 2% of the ETF; the company is benefiting from surging AI processing demand and is expected to see significant sales growth with large orders on the horizon.
- Market Reaction Analysis: Although the performance of Ark Innovation ETF has been mixed, Cathie Wood's continued investment in these high-risk stocks reflects her strong confidence in the innovation sector, which may significantly impact the fund's overall performance in the coming years.
See More
CRISPR Therapeutics Shares Surge on Strong Earnings and Pipeline Growth
- Significant Revenue Growth: CRISPR Therapeutics' gene-editing therapy Casgevy generated $54 million in Q4 2025 and $116 million for the full year, indicating strong market demand for treatments of sickle cell disease and transfusion-dependent beta thalassemia.
- Surge in Patient Treatments: In Q4, 64 patients received Casgevy infusions, with 147 initiating treatment in the first quarter of 2025, nearly tripling 2024 levels, suggesting that successful patient recruitment will drive substantial revenue growth in the future.
- Analyst Optimism: William Blair analyst Sami Corwin noted that the increase in first cell collections strengthens confidence in significantly higher revenue expectations for 2026, reflecting market confidence in CRISPR's future performance.
- Positive Technical Analysis: CRISPR Therapeutics' stock is currently trading 14.4% above its 20-day SMA and 3.0% above its 100-day SMA, indicating a strong short-term upward trend, while the stock has risen 33.31% over the past 12 months, approaching its 52-week highs.
See More
Crispr Therapeutics Rises 5% Amid Acquisition Speculation
- Acquisition Speculation: Crispr Therapeutics (CRSP) shares surged 5% amid rising acquisition speculation, indicating strong market optimism that could attract further investor interest in the company.
- Vertex Pharmaceuticals Involvement: Traders noted that Vertex Pharmaceuticals (VRTX) has been considering acquiring Crispr since late last year, highlighting major pharmaceutical interest in gene-editing technologies and potential industry consolidation.
- Legal Counsel Engagement: Reports suggest that Vertex has engaged Skadden law firm to assist in evaluating the acquisition, reflecting the seriousness of its intentions towards Crispr and potentially accelerating negotiation processes.
- Significant Market Potential: Betaville reported in November that a US-based biopharma with a market cap of $100-$200 billion may be interested in bidding for Crispr, showcasing confidence in the long-term growth potential of gene therapies.
See More
FDA Unveils New Guidance to Accelerate Rare Disease Therapy Approvals
- Accelerated Approval Process: The FDA's draft guidance aims to expedite the approval of therapies for rare diseases, particularly focusing on individualized genome editing and RNA-based therapies, which is expected to significantly shorten time to market and enhance R&D enthusiasm among companies.
- Clear Treatment Focus: The new guidance emphasizes treatments targeting specific genetic, cellular, or molecular abnormalities, requiring sponsors to identify disease-causing abnormalities and demonstrate that therapies effectively target the underlying causes, thereby promoting the development of more targeted treatment options.
- Clinical Trial Flexibility: The FDA allows for the evaluation of different product variants targeting mutations in a single gene within a single product application, utilizing master protocols to assess these variants in one trial, which enhances the efficiency and flexibility of clinical trials.
- Beneficial Companies: Several companies, including Ultragenyx, Regenxbio, and CRISPR Therapeutics, could leverage the new framework, which is expected to drive innovation and competitiveness in the rare disease sector, further promoting growth in the biopharmaceutical industry.
See More
CRISPR Therapeutics: Potential and Challenges in Gene Editing
- Therapeutic Progress: CRISPR's gene-editing therapy Casgevy was approved in 2023 for treating sickle cell disease and transfusion-dependent beta thalassemia, showcasing its revolutionary potential in healthcare.
- High Treatment Costs: Despite Casgevy's staggering price of $2.2 million, healthcare experts justify this cost due to its one-time treatment nature, which could save patients time and money in the long run.
- Financial Challenges: CRISPR reported a net loss of $581.6 million in 2025, an increase from the $366.3 million loss in 2024, highlighting the significant expenditure pressures the company faces as it rolls out Casgevy.
- Strong Cash Position: As of the end of last year, CRISPR had approximately $2 billion in cash and marketable securities, indicating that despite its losses, the company is not at immediate risk of running out of funds, maintaining long-term growth potential.
See More
CRISPR Therapeutics Faces Significant Challenges Amid Slow Rollout
- Escalating Financial Losses: CRISPR reported a net loss exceeding $580 million in 2025, a significant increase from the $366.3 million loss in 2024, highlighting the pressure from high expenditures during the rollout of the Casgevy treatment and ongoing financial struggles.
- Strong Cash Position: Despite the losses, CRISPR maintained approximately $2 billion in cash and marketable securities at the end of last year, which is sufficient to support its daily operations, having consumed $345 million in 2025, indicating that the company is not at immediate risk of running out of funds.
- Slow Product Rollout: Although Casgevy was approved in 2023 and patient initiations nearly tripled last year, its staggering price of $2.2 million and profit-sharing with Vertex Pharmaceuticals limit CRISPR's market earnings, adversely affecting its profitability.
- Uncertain Market Outlook: While CRISPR's gene-editing therapies hold long-term potential, the lack of current profitability and the risk assessment of its stock make it a cautious buy for investors, especially as it was not included in the list of top recommended stocks by analysts.
See More
Cathie Wood's Latest Investment Moves in Ark Innovation ETF
- Figma Investment Update: Ark Innovation ETF purchased additional shares of Figma on February 23, making it the 38th largest holding at 0.66% of the total portfolio; despite concerns about slowing growth, Figma's 40% year-over-year sales increase in Q4 indicates strong market potential.
- CRISPR Holdings Overview: On February 20, Ark increased its stake in CRISPR Therapeutics, now the second-largest holding at 6.64% of the fund, with only $3.5 million in sales last year, yet the potential commercialization of gene-editing drugs could lead to explosive growth, representing a high-risk, high-reward investment.
- CoreWeave Investment Outlook: Ark also bought more shares of CoreWeave on February 20, making it the 18th largest holding at approximately 2% of the ETF; the company is benefiting from surging AI processing demand and is expected to see significant sales growth with large orders on the horizon.
- Market Reaction Analysis: Although the performance of Ark Innovation ETF has been mixed, Cathie Wood's continued investment in these high-risk stocks reflects her strong confidence in the innovation sector, which may significantly impact the fund's overall performance in the coming years.
See More
CRISPR Therapeutics Shares Surge on Strong Earnings and Pipeline Growth
- Significant Revenue Growth: CRISPR Therapeutics' gene-editing therapy Casgevy generated $54 million in Q4 2025 and $116 million for the full year, indicating strong market demand for treatments of sickle cell disease and transfusion-dependent beta thalassemia.
- Surge in Patient Treatments: In Q4, 64 patients received Casgevy infusions, with 147 initiating treatment in the first quarter of 2025, nearly tripling 2024 levels, suggesting that successful patient recruitment will drive substantial revenue growth in the future.
- Analyst Optimism: William Blair analyst Sami Corwin noted that the increase in first cell collections strengthens confidence in significantly higher revenue expectations for 2026, reflecting market confidence in CRISPR's future performance.
- Positive Technical Analysis: CRISPR Therapeutics' stock is currently trading 14.4% above its 20-day SMA and 3.0% above its 100-day SMA, indicating a strong short-term upward trend, while the stock has risen 33.31% over the past 12 months, approaching its 52-week highs.
See More
Crispr Therapeutics Rises 5% Amid Acquisition Speculation
- Acquisition Speculation: Crispr Therapeutics (CRSP) shares surged 5% amid rising acquisition speculation, indicating strong market optimism that could attract further investor interest in the company.
- Vertex Pharmaceuticals Involvement: Traders noted that Vertex Pharmaceuticals (VRTX) has been considering acquiring Crispr since late last year, highlighting major pharmaceutical interest in gene-editing technologies and potential industry consolidation.
- Legal Counsel Engagement: Reports suggest that Vertex has engaged Skadden law firm to assist in evaluating the acquisition, reflecting the seriousness of its intentions towards Crispr and potentially accelerating negotiation processes.
- Significant Market Potential: Betaville reported in November that a US-based biopharma with a market cap of $100-$200 billion may be interested in bidding for Crispr, showcasing confidence in the long-term growth potential of gene therapies.
See More
FDA Unveils New Guidance to Accelerate Rare Disease Therapy Approvals
- Accelerated Approval Process: The FDA's draft guidance aims to expedite the approval of therapies for rare diseases, particularly focusing on individualized genome editing and RNA-based therapies, which is expected to significantly shorten time to market and enhance R&D enthusiasm among companies.
- Clear Treatment Focus: The new guidance emphasizes treatments targeting specific genetic, cellular, or molecular abnormalities, requiring sponsors to identify disease-causing abnormalities and demonstrate that therapies effectively target the underlying causes, thereby promoting the development of more targeted treatment options.
- Clinical Trial Flexibility: The FDA allows for the evaluation of different product variants targeting mutations in a single gene within a single product application, utilizing master protocols to assess these variants in one trial, which enhances the efficiency and flexibility of clinical trials.
- Beneficial Companies: Several companies, including Ultragenyx, Regenxbio, and CRISPR Therapeutics, could leverage the new framework, which is expected to drive innovation and competitiveness in the rare disease sector, further promoting growth in the biopharmaceutical industry.
See More
