[CRISPR Therapeutics Business Update and Q2 2025 Financial Results](index=1&type=section&id=CRISPR%20Therapeutics%20Business%20Update%20and%20Q2%202025%20Financial%20Results) [Business Highlights and Outlook](index=1&type=section&id=Business%20Highlights%20and%20Outlook) The company reports strong commercial and clinical program momentum, with key data updates expected and a solid cash position of approximately $1.7 billion - The company is experiencing momentum across its commercial and clinical programs, with several key milestones anticipated in the **second half of 2025**[2](index=2&type=chunk) - Maintains a strong balance sheet with approximately **$1.7 billion in cash**, cash equivalents, and marketable securities as of June 30, 2025[1](index=1&type=chunk) [Hemoglobinopathies and CASGEVY®](index=1&type=section&id=Hemoglobinopathies%20and%20CASGEVY%C2%AE) The commercial launch of CASGEVY® is progressing with 75 treatment centers activated globally and reimbursement agreements secured in 10 countries - Achieved the target of activating **75 authorized treatment centers (ATCs)** globally for CASGEVY®[3](index=3&type=chunk) - As of June 30, approximately **115 patients have completed cell collection**, and 29 patients have received CASGEVY® infusions, with 16 of those occurring in Q2[3](index=3&type=chunk) - Vertex has secured **reimbursement agreements for eligible patients in 10 countries**, including recent additions of Northern Ireland, Scotland, and Denmark[6](index=6&type=chunk) - Advancing next-generation approaches, including an anti-CD117 ADC and an in vivo editing platform, to expand the addressable patient population[6](index=6&type=chunk) [Immuno-Oncology and Autoimmune Disease Programs](index=2&type=section&id=Immuno-Oncology%20and%20Autoimmune%20Disease%20Programs) The company is advancing its next-generation CAR T candidates, with CTX112™ receiving RMAT designation and broad data updates expected in H2 2025 - CTX112, targeting CD19, received **Regenerative Medicine Advanced Therapy (RMAT) designation** from the FDA for relapsed or refractory follicular lymphoma and marginal zone lymphoma[6](index=6&type=chunk) - A Phase 1 trial of CTX112 is ongoing for autoimmune indications, including **systemic lupus erythematosus (SLE)**[6](index=6&type=chunk) - Broad updates for CTX112 in oncology and autoimmune disease are expected in **H2 2025**, with updates for the CTX131 program also expected in 2025[1](index=1&type=chunk)[6](index=6&type=chunk) [In Vivo Liver Editing Programs](index=2&type=section&id=In%20Vivo%20Liver%20Editing%20Programs) The in vivo pipeline shows progress with CTX310™ demonstrating significant reductions in triglycerides and LDL, with a full data presentation expected in H2 2025 CTX310™ Preliminary Phase 1 Data Highlights | Metric | Peak Reduction | | :--- | :--- | | Triglycerides (TG) | up to 82% | | Low-density lipoprotein (LDL) | up to 86% | - The company anticipates presenting the complete Phase 1 data for CTX310 at a medical meeting in the **second half of 2025**[1](index=1&type=chunk)[10](index=10&type=chunk) - A clinical trial for CTX320™, targeting the LPA gene, is ongoing, with an update expected in the **first half of 2026**[1](index=1&type=chunk)[10](index=10&type=chunk) [SRSD107 and Sirius Therapeutics Collaboration](index=4&type=section&id=SRSD107%20and%20Sirius%20Therapeutics%20Collaboration) A strategic collaboration with Sirius Therapeutics was initiated to co-develop siRNA therapies, with SRSD107 receiving EMA authorization for a Phase 2 trial - Entered a strategic collaboration with Sirius Therapeutics to jointly develop and commercialize siRNA therapies, beginning with SRSD107[10](index=10&type=chunk) - Received European Medicines Agency (EMA) authorization to initiate a **Phase 2 clinical trial** of SRSD107 for thromboembolic disorders[1](index=1&type=chunk)[10](index=10&type=chunk) [Regenerative Medicine Programs](index=4&type=section&id=Regenerative%20Medicine%20Programs) The company is advancing its regenerative medicine programs for Type 1 Diabetes, with an update on next-generation therapies expected in 2025 - Developing next-generation programs for Type 1 Diabetes (T1D) using iPSC-derived, allogeneic, gene-edited beta islet cell precursors to enable **insulin independence without immunosuppression**[9](index=9&type=chunk) - An update on the regenerative medicine programs is expected in **2025**[9](index=9&type=chunk) [Second Quarter 2025 Financial Results](index=5&type=section&id=Second%20Quarter%202025%20Financial%20Results) The company reported a net loss of $208.5 million for Q2 2025, driven by a one-time R&D expense, while maintaining a strong cash position of $1.72 billion [Financial Performance Summary](index=5&type=section&id=Financial%20Performance%20Summary) The company's cash position decreased to $1.72 billion, while net loss increased to $208.5 million due to a $96.3 million in-process R&D expense Q2 2025 Financial Highlights (vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | Change Driver | | :--- | :--- | :--- | :--- | | Cash Position (June 30) | $1,721.2M | N/A | Decrease from Dec 31, 2024 ($1,903.8M) due to operating expenses and Sirius payment | | R&D Expenses | $69.9M | $80.2M | Decrease in employee-related expenses | | Acquired In-Process R&D | $96.3M | $0 | Costs incurred from Sirius Agreement | | G&A Expenses | $18.9M | $19.5M | Remained relatively stable | | Collaboration Expense, net | $45.2M | $52.1M | Decrease due to higher CASGEVY revenue and lower program expenses | | Net Loss | ($208.5M) | ($126.4M) | Increased primarily due to acquired in-process R&D expense | [Condensed Consolidated Statements of Operations](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) For Q2 2025, the company reported total revenues of $0.89 million and a net loss of $208.5 million, or ($2.40) per share Condensed Consolidated Statements of Operations (Unaudited, In thousands) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | **Total revenue** | **$892** | **$517** | **$1,757** | **$1,021** | | Research and development | $69,894 | $80,165 | $142,378 | $156,338 | | Acquired in-process R&D | $96,253 | $— | $96,253 | $— | | General and administrative | $18,916 | $19,481 | $38,212 | $37,434 | | Collaboration expense, net | $45,153 | $52,131 | $102,662 | $99,097 | | **Total operating expenses** | **$230,216** | **$151,777** | **$379,505** | **$292,869** | | **Loss from operations** | **($229,324)** | **($151,260)** | **($377,748)** | **($291,848)** | | **Net loss** | **($208,549)** | **($126,408)** | **($344,545)** | **($242,999)** | | **Net loss per common share — basic** | **($2.40)** | **($1.49)** | **($3.98)** | **($2.92)** | [Condensed Consolidated Balance Sheets Data](index=8&type=section&id=Condensed%20Consolidated%20Balance%20Sheets%20Data) As of June 30, 2025, the company held $1.72 billion in cash and marketable securities, with total assets of $2.03 billion Condensed Consolidated Balance Sheets Data (Unaudited, in thousands) | | As of June 30, 2025 | As of December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $193,618 | $298,257 | | Marketable securities | $1,527,619 | $1,605,569 | | Working capital | $1,629,304 | $1,849,350 | | Total assets | $2,029,711 | $2,242,034 | | Total shareholders' equity | $1,711,125 | $1,932,080 |

Core Insights - CRISPR Therapeutics is experiencing significant momentum in both commercial and clinical programs, particularly with the activation of over 75 authorized treatment centers for CASGEVY, which has led to approximately 115 patients having cells collected globally [1][2][3] - The company is advancing multiple clinical trials, including CTX310 targeting ANGPTL3, which has shown promising preliminary data with reductions of up to 82% in triglycerides and 86% in LDL [1][3][7] - CRISPR Therapeutics has a strong financial position with approximately $1.7 billion in cash and marketable securities as of June 30, 2025, despite a net loss of $208.5 million for the second quarter of 2025 [1][14][29] Commercial Programs - CASGEVY, a CRISPR/Cas9 gene-edited cell therapy, is approved in multiple regions including the U.S., EU, and Canada for treating sickle cell disease and transfusion-dependent beta thalassemia [3][12] - The company has achieved its target of activating 75 authorized treatment centers globally, with 29 patients having received infusions of CASGEVY, including 16 in the second quarter of 2025 [1][3] - Reimbursement agreements have been secured in 10 countries, enhancing patient access to CASGEVY [3] Clinical Trials - Ongoing clinical trials for CTX310™ are expected to present complete Phase 1 data in the second half of 2025, with preliminary results indicating significant reductions in triglycerides and LDL [1][3][7] - CTX320™ is in a Phase 1 trial targeting the LPA gene, with updates anticipated in the first half of 2026 [1][3] - CTX112™ and CTX131™ are also in clinical trials targeting CD19 and CD70, respectively, with broad updates expected in the second half of 2025 [1][3][7] Financial Performance - For the second quarter of 2025, CRISPR Therapeutics reported a net loss of $208.5 million, compared to a net loss of $126.4 million in the same period of 2024 [14][29] - Research and development expenses decreased to $69.9 million from $80.2 million year-over-year, primarily due to reduced employee-related costs [14][29] - The total operating expenses for the second quarter were $230.2 million, up from $151.8 million in the previous year, largely due to acquired in-process R&D expenses related to the Sirius Agreement [14][29] Strategic Collaborations - A strategic collaboration with Sirius Therapeutics was established to develop and commercialize small interfering RNA (siRNA) therapies, starting with SRSD107 for thromboembolic disorders [13][20] - The European Medicines Agency has authorized a Phase 2 clinical trial for SRSD107, which aims to evaluate its safety and efficacy in preventing postoperative venous thromboembolism [13][20]

Group 1 - CRISPR Therapeutics AG (CRSP) has an average brokerage recommendation (ABR) of 1.88, indicating a consensus between Strong Buy and Buy based on 27 brokerage firms' recommendations [2][5] - Out of the 27 recommendations, 15 are classified as Strong Buy, accounting for 55.6%, while one is classified as Buy, accounting for 3.7% [2] - Despite the positive ABR, reliance solely on brokerage recommendations may not be advisable, as studies suggest they often fail to guide investors effectively towards stocks with high price appreciation potential [5][10] Group 2 - Brokerage analysts tend to exhibit a strong positive bias in their ratings due to vested interests, often resulting in a disproportionate number of Strong Buy recommendations compared to Strong Sell [6][10] - The Zacks Rank, a proprietary stock rating tool, is presented in whole numbers and is based on earnings estimate revisions, which have shown a strong correlation with near-term stock price movements [8][11] - The Zacks Consensus Estimate for CRISPR Therapeutics has declined by 1.4% over the past month to -$5.61, indicating growing pessimism among analysts regarding the company's earnings prospects [14][15] Group 3 - The recent change in the consensus estimate, along with other earnings-related factors, has led to a Zacks Rank of 4 (Sell) for CRISPR Therapeutics, suggesting caution despite the favorable ABR [15] - The ABR may not always reflect the most current information, while the Zacks Rank is updated more frequently to reflect changes in earnings estimates [13]

Company Overview - CRISPR Therapeutics AG (CRSP) is expected to report a quarterly loss of $1.47 per share, reflecting a year-over-year change of +1.3% [3] - Revenues are anticipated to be $6.58 million, representing a significant increase of 1165.4% from the same quarter last year [3] Earnings Estimates and Revisions - The consensus EPS estimate has been revised 1.75% lower over the last 30 days, indicating a reassessment by analysts [4] - The Most Accurate Estimate for CRISPR Therapeutics is lower than the Zacks Consensus Estimate, resulting in an Earnings ESP of -11.09% [12] Earnings Surprise Prediction - A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a favorable Zacks Rank [10] - CRISPR Therapeutics currently holds a Zacks Rank of 4, which complicates the prediction of an earnings beat [12] Historical Performance - In the last reported quarter, CRISPR Therapeutics was expected to post a loss of $1.27 per share but actually reported a loss of -$1.58, resulting in a surprise of -24.41% [13] - Over the past four quarters, the company has beaten consensus EPS estimates two times [14] Industry Context - Another player in the biomedical and genetics industry, Illumina (ILMN), is expected to post earnings of $1.02 per share, indicating a year-over-year change of +183.3% [18] - Illumina's revenues are projected to be $1.05 billion, down 5.8% from the previous year, with a slight downward revision of 0.2% in the consensus EPS estimate over the last 30 days [19]

Group 1 - CRISPR Therapeutics AG (CRSP) has an average brokerage recommendation (ABR) of 1.88, indicating a consensus between Strong Buy and Buy based on 27 brokerage firms' recommendations [2] - Out of the 27 recommendations, 15 are classified as Strong Buy, accounting for 55.6%, while one is classified as Buy, accounting for 3.7% [2] - Despite the positive ABR, caution is advised as studies show limited success of brokerage recommendations in predicting stock price increases [5][10] Group 2 - Brokerage analysts tend to exhibit a strong positive bias in their ratings due to vested interests, with five Strong Buy recommendations for every Strong Sell [6][10] - The Zacks Rank, a proprietary stock rating tool, is highlighted as a more reliable indicator of near-term price performance, based on earnings estimate revisions [8][11] - The Zacks Consensus Estimate for CRISPR Therapeutics remains unchanged at -$5.54, suggesting the stock may perform in line with the broader market [13][14] Group 3 - The Zacks Rank for CRISPR Therapeutics is currently 3 (Hold), indicating a more cautious stance compared to the Buy-equivalent ABR [14] - The ABR is not always up-to-date, while the Zacks Rank reflects timely earnings estimate revisions, making it a more current indicator of future price movements [12]

Core Insights - The article discusses the author's transition to independent investment research after over 43 years in the industry, emphasizing a focus on actionable investment insights rather than adhering to external agendas [1] - The author specializes in rules and factor-based equity investing strategies, combining quantitative analysis with fundamental analysis to derive investment stories that predict future performance [1] - The author has extensive experience across various market segments, including large cap, small cap, micro cap, value, growth, and income stocks, as well as managing a high-yield fixed-income fund [1] Group 1 - The author aims to provide the best actionable investment insights without being influenced by any product agenda [1] - The approach taken is to use numbers as a tool to inspire human intelligence-driven investment narratives rather than relying solely on statistical studies [1] - The author has a background in investor education, having conducted seminars and authored books on stock selection and analysis [1] Group 2 - The author has developed and worked with various quantitative models and has experience in quantitative asset allocation strategies, which are foundational to modern Robo Advising [1] - The article highlights the author's previous roles, including editing stock newsletters and serving as an assistant research director at Value Line [1] - The author expresses openness to feedback and criticism from readers, indicating a willingness to engage with the investment community [1]

Group 1: Stock Performance - CRISPR Therapeutics AG shares increased by 9.6% to close at $60.08, with notable trading volume compared to typical sessions [1] - The stock has gained 27.7% over the past four weeks, indicating strong investor interest [1] Group 2: Pipeline Developments - The rise in CRISPR's stock price is attributed to positive sentiment regarding its pipeline, particularly the in vivo candidate CTX310 targeting ANGPTL3 for atherosclerotic heart disease [2] - Updated data from an early-stage study showed a single dose of CTX310 resulted in peak reductions of up to 82% in triglyceride levels and up to 86% in LDL levels, generating excitement for other pipeline candidates [2] Group 3: Financial Expectations - The company is expected to report a quarterly loss of $1.54 per share, reflecting a year-over-year change of -3.4%, while revenues are projected at $5.89 million, a significant increase of 1032.9% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, suggesting that stock price movements may be influenced by trends in earnings estimate revisions [4] Group 4: Industry Context - CRISPR Therapeutics is part of the Zacks Medical - Biomedical and Genetics industry, where Wave Life Sciences also operates, closing the last trading session 10.8% higher at $7.7 [4] - Wave Life Sciences has seen a consensus EPS estimate change of -6.6% over the past month, indicating a potential decline in performance compared to the previous year [5]

Company Performance - CRISPR Therapeutics AG (CRSP) ended the recent trading session at $51.19, showing a -1.88% change from the previous day's closing price, which lagged behind the S&P 500's daily loss of 0.79% [1] - The stock has risen by 24.63% in the past month, outperforming the Medical sector's gain of 1.49% and the S&P 500's gain of 5.22% [1] Financial Forecast - The company is forecasted to report an EPS of -$1.54, reflecting a 3.36% decrease from the same quarter of the previous year [2] - Quarterly revenue is expected to be $5.89 million, representing a significant increase of 1032.88% from the year-ago period [2] Full-Year Estimates - Full-year Zacks Consensus Estimates predict earnings of -$5.54 per share and revenue of $39.95 million, indicating year-over-year changes of -27.65% and +7.06%, respectively [3] - Recent changes to analyst estimates for CRISPR Therapeutics AG can indicate short-term business trends, with positive revisions seen as a favorable sign for the business outlook [3] Zacks Rank and Industry Performance - The Zacks Rank system, which includes estimate changes, has a proven track record of outperformance, with 1 stocks returning an average of +25% annually since 1988 [5] - CRISPR Therapeutics AG currently holds a Zacks Rank of 3 (Hold), with the consensus EPS estimate remaining stagnant over the past month [5] - The Medical - Biomedical and Genetics industry, part of the Medical sector, has a Zacks Industry Rank of 77, placing it within the top 32% of over 250 industries [6]

Group 1: CRISPR Therapeutics - CRISPR Therapeutics has developed Casgevy, the first gene-editing medicine approved using the CRISPR technique, but the stock has been on a downward trend since 2021 due to clinical progress, complexity of therapy administration, and unprofitability [3][4] - The company is targeting challenging areas such as type 1 diabetes and hard-to-treat cancers, with expected data readouts for ongoing clinical trials as early as this year, which could positively impact stock performance [5] - Casgevy has significant financial implications, with a treatment cost of $2.2 million in the U.S. and an estimated 60,000 patients in target geographies, indicating blockbuster potential [9] Group 2: Viking Therapeutics - Viking Therapeutics gained attention after strong phase 2 results for VK2735, an investigational weight management therapy, but has seen stock performance decline as investors took profits [10] - The anti-obesity therapy market is rapidly growing, and Viking's VK2735 has shown promising mid-stage data, with an oral formulation currently in Phase 2 studies [11] - Viking Therapeutics has a robust pipeline, including VK2809 for metabolic dysfunction-associated steatohepatitis entering phase 3 studies and VK0214 for X-linked adrenoleukodystrophy, which has received orphan drug designation [12][13]

Core Insights - The healthcare sector is highlighted as having significant growth opportunities, particularly in the weight loss drug market and gene editing technologies [2][4]. Group 1: Weight Loss Drug Market - Eli Lilly is positioned to potentially lead the GLP-1 agonist market, currently holding approximately 35% market share, while Novo Nordisk leads with 65% [4]. - The GLP-1 market is projected to grow to a $150 billion opportunity over the next decade, representing a tenfold increase from last year's sales [4]. - Eli Lilly is developing two promising drugs: Orforglipron, an oral GLP-1 pill, and Retatrutide, which targets multiple hormones and is in phase 3 studies [5][6]. - Analysts anticipate Eli Lilly will achieve 32% annualized earnings growth over the long term, despite a high price-to-earnings (P/E) ratio of nearly 65 [7][8]. Group 2: Gene Editing Technology - CRISPR Therapeutics is advancing its commercialization efforts for Casgevy, a therapy for sickle cell disease and beta-thalassemia, which is the first CRISPR-based treatment to receive FDA approval [10]. - The company has five additional therapies in clinical trials, indicating potential for significant growth if successful [11]. - Analysts project CRISPR Therapeutics will achieve $173 million in revenue next year, with an enterprise value of $2.2 billion, suggesting a reasonable price for the stock given its potential [12].