Key Takeaways CRSP is advancing CTX310 and CTX320 in early trials targeting heart disease-linked genetic factors. CTX310 showed up to 82% TG and 81% LDL reduction in early dose-dependent trial results. CRSP plans to add two more in vivo programs by year-end.CRISPR Therapeutics (CRSP) is currently the only company in the world to have secured approval for a CRISPR-based gene therapy developed in partnership with Vertex Pharmaceuticals. Following the landmark success with Casgevy, an ex vivo therapy, the co ...

CRISPR Therapeutics AG (NASDAQ:CRSP) William Blair’s 45th Annual Growth Stock Conference June 3, 2025 12:20 PM ET Company Participants Raju Prasad - Chief Financial Officer Conference Call Participants Sami Corwin - William Blair Sami Corwin Hi, everyone. We're going to get started. I'm Sami Corwin, the biotech equity research analyst covering CRISPR at William Blair. Before we get started, I would like to direct everyone to williamblair.com for a full list of disclosures. I'm very pleased to introduce CRIS ...

Summary of CRISPR Therapeutics (CRSP) FY Conference Call - June 03, 2025 Company Overview - CRISPR Therapeutics is focused on creating transformative gene-based medicines for serious diseases, leveraging its Nobel Prize-winning CRISPR technology [3][4][32]. Key Products and Pipeline - **KASJEVY**: The flagship product for treating sickle cell disease, with a global addressable market of approximately 60,000 patients and a price point of $2 million, creating a multibillion-dollar market opportunity [10][11]. - **Clinical Trials**: Several ongoing clinical trials with readouts expected in the next 6 to 12 months, which will shape the company's strategic direction [4][8]. - **Franchises**: The company has four key franchises: 1. **Hematology**: KASJEVY as the anchor product. 2. **CAR T Platform**: Targeting oncology and autoimmune diseases using CRISPR technology [5][14]. 3. **In Vivo Platform**: Developing therapies for cardiovascular diseases, including a one-time infusion to reduce triglycerides and LDL cholesterol [6][17]. 4. **Type 1 Diabetes**: Aiming to provide insulin-producing pancreatic islet cells [6][28]. Financial Highlights - Strong balance sheet with $1.86 billion in cash, allowing for purposeful spending and investment in growth opportunities [8][31][50]. - Anticipation of significant revenue generation from KASJEVY and other pipeline products, with a focus on achieving profitability in the future [49][50]. Recent Developments - **Partnership with Vertex**: Vertex is leading the commercialization of KASJEVY, with CRISPR receiving 40% of the net income from the program [31][45]. - **Business Development Deal**: Acquisition of siRNA technology from Sirius Therapeutics targeting Factor XI, which presents a multibillion-dollar opportunity in the anticoagulation space [8][31][33]. Clinical Data and Efficacy - **CTX-310**: Early data shows a 56% average reduction in triglycerides and a 28% reduction in LDL cholesterol, with some patients experiencing reductions of up to 82% and 65%, respectively [19][20][21]. - **CTX-112**: In oncology trials, a 67% overall response rate and a 50% complete response rate were reported, indicating promising efficacy [15][16]. Market Opportunities - Expansion into untapped markets, particularly in the Middle East, with significant patient populations for KASJEVY [12][48]. - Potential for addressing underserved populations in Africa and other regions with innovative therapies [13][45]. Strategic Vision - CRISPR aims to become a sector-leading biotech company with a sustainable business model, focusing on innovation and the development of a diverse pipeline [8][32][40]. - The company is committed to maintaining a high hurdle rate for new assets and partnerships, ensuring that any new initiatives align with its long-term goals [39][40]. Conclusion - 2025 is viewed as a pivotal year for CRISPR Therapeutics, with multiple clinical data readouts and the ongoing launch of KASJEVY expected to drive growth and establish the company as a leader in the biotech sector [31][32].

ZUG, Switzerland and BOSTON, May 29, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that members of its senior management team are scheduled to participate in the following investor conferences in June. William Blair’s 45th Annual Growth Stock ConferenceDate: Tuesday, June 3, 2025Time: 11:20 a.m. CT Goldman Sachs’ 46th Annual Global Healthcare ConferenceDate: Monday, June 9 ...

Financial Data and Key Metrics Changes - The company reported a solid financial position with $1.6 billion on the balance sheet, indicating strong capital efficiency and the ability to fund future projects [3][4]. Business Line Data and Key Metrics Changes - The approved product, Kasjevi, is now in eight different jurisdictions for treating sickle cell and beta thalassemia, with a revenue of $14 million generated from 90 cells collected and 65 ATCs as of Q1 [12][13][22]. - The company is targeting 75 ATCs to support the demand for Kasjevi, indicating a significant multi-billion dollar market opportunity [14][16]. Market Data and Key Metrics Changes - The company sees a substantial market in the Middle East, with over 23,000 patients identified in Gulf Coast countries, which is expected to play a significant role in Kasjevi's market expansion [16][17]. Company Strategy and Development Direction - The company aims to focus on transformative gene-based medicines while exploring opportunistic deals, such as the partnership with Sirius Therapeutics for an siRNA platform [4][8]. - The strategic approach includes a phase two trial for Factor XI, which is expected to provide immediate value-creating catalysts in the next 12 to 18 months [10][11]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the launch performance of Kasjevi and the potential for significant patient initiation growth, supported by manufacturing investments [13][14][22]. - The company is optimistic about the upcoming data readouts from various programs, including CTX 310, which showed promising early results with an 82% reduction in triglycerides and 81% reduction in LDL [6][28]. Other Important Information - The company is actively looking for business development opportunities in a buyer's market, with plans to explore partnerships that can create significant value [48][50]. - The cost of goods for new therapies is expected to be in the low five-figure range, allowing for competitive pricing against existing therapies [44][45]. Q&A Session Summary Question: What does the Sirius deal do for your platform with siRNAs? - The Sirius deal is seen as a diversification play, allowing the company to explore complementary assets in a buyer's market [8][9]. Question: How has the launch of Kasjevi been trending? - The launch has been successful, with significant revenue and patient initiation growth expected [12][13]. Question: What are the key days to watch for the Factor XI program? - The initiation of the TKA study in the second half of the year is a key milestone [11]. Question: How does the company plan to address the preconditioning regimen for Kasjevi? - The company is working on approaches to make the preconditioning regimen more comfortable for patients [23][25]. Question: What is the company's strategy for broad diseases versus rare diseases? - The company aims to ensure low production costs to remain competitive in both broad and rare disease markets [44][45].

Core Insights - CRISPR Therapeutics has entered a collaboration with Sirius Therapeutics to develop and commercialize novel siRNA therapies, specifically targeting thromboembolic disorders [1][3] - The lead candidate from this partnership, SRSD107, has shown promising results in early studies, indicating potential as a best-in-class therapy for thromboembolism [5][6] Financial Terms of the Collaboration - CRISPR Therapeutics will make an upfront cash payment of $25 million and $70 million in equity to Sirius Therapeutics, with both companies sharing development costs and profits equally [3][4] - CRISPR will handle commercialization in the U.S., while Sirius will lead in Greater China [3] Development Status of SRSD107 - Phase I studies have demonstrated over 93% peak reductions in FXI levels and activity, with a significant increase in activated partial thromboplastin time [5] - A Phase II study has been initiated to evaluate the safety and efficacy of SRSD107 for preventing venous thromboembolism in patients undergoing total knee arthroplasty [6] Recent Achievements - CRISPR Therapeutics, in partnership with Vertex Pharmaceuticals, received approval for Casgevy, a CRISPR/Cas9 gene-edited therapy for sickle cell disease and transfusion-dependent beta thalassemia, marking a significant milestone in medical science [8] - Vertex leads the global development and commercialization of Casgevy, sharing costs and profits with CRISPR in a 60:40 ratio [9]

Independent drug launches are highly unpredictable, but CRISPR Therapeutics can lean on its collaboration partner, Vertex Pharmaceuticals (VRTX 0.40%). Vertex successfully markets the first and only FDA-approved treatments that address the underlying cause of cystic fibrosis, another rare disease. With product sales that grew past $11 billion last year, it's clear Vertex knows a thing or two about selling rare disease drugs. CRISPR Therapeutics is still losing money, but it also finished March with $1.9 bil ...

Core Insights - CRISPR Therapeutics and Sirius Therapeutics have formed a strategic partnership to co-develop and commercialize SRSD107, a long-acting Factor XI (FXI) small interfering RNA (siRNA) for thromboembolic disorders [1][2] - SRSD107 has shown promising results in Phase 1 clinical trials, achieving over 93% reduction in FXI activity and more than twofold increase in activated partial thromboplastin time (aPTT), with effects lasting up to 6 months post-dosing [1][3] - The collaboration includes an upfront payment of $25 million in cash and $70 million in equity from CRISPR to Sirius, with a 50-50 cost and profit-sharing structure for SRSD107 [1][8] Company Overview - CRISPR Therapeutics is focused on creating transformative gene-based medicines and has a diverse portfolio across various disease areas, including cardiovascular diseases [10] - Sirius Therapeutics is a clinical-stage biotech company developing innovative siRNA therapies, with SRSD107 being its most advanced product targeting thromboembolic disorders [17][18] Clinical Development - The clinical program for SRSD107 includes two Phase 1 trials demonstrating safety and robust pharmacodynamic effects, with a Phase 2 trial planned to evaluate its efficacy in preventing venous thromboembolism (VTE) in total knee arthroplasty patients [3][7] - SRSD107 is designed to selectively inhibit FXI, aiming to reduce thrombotic events while minimizing bleeding risks, offering a differentiated approach compared to existing therapies [2][16] Market Potential - Thromboembolic disorders are a significant global health issue, affecting millions and representing a substantial unmet medical need [2][15] - The addressable patient population for SRSD107 includes those with atrial fibrillation, venous thromboembolism, cancer-associated thrombosis, and other conditions where existing therapies pose bleeding risks [2][3]

Core Viewpoint - CRISPR Therapeutics has faced financial struggles despite clinical advancements, with shares down 24% over the past three years, while the S&P 500 gained 41%. However, potential catalysts could double its stock price by 2030, indicating a compound annual growth rate of about 14.9% [1] Group 1: Clinical Progress and Products - CRISPR Therapeutics has developed Casgevy, a one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia, showcasing the potential of its gene-editing technology [2] - The complexity of ex vivo gene-editing treatments, which involve collecting, editing, and reinserting patient cells, presents manufacturing and administration challenges [3] - Although Casgevy was approved in late 2023, it has not yet significantly impacted financial results, but it is expected to exceed $1 billion in annual sales at its peak due to limited competition [5][6] Group 2: Market Potential - The addressable market for Casgevy is estimated at 58,000 patients in targeted regions, with a treatment cost of $2.2 million in the U.S., indicating substantial revenue potential [6] - The competitive landscape for CRISPR and Vertex Pharmaceuticals appears favorable, particularly in the Middle East where they have received product approvals [5] Group 3: Future Catalysts - CRISPR Therapeutics has several promising candidates in its pipeline, including CTX112 and CTX131 for cancer, with CTX112 receiving Regenerative Medicine Advanced Therapy designation from the FDA [10] - Data from an ongoing clinical trial for CTX320, aimed at lowering lipoprotein(a) levels, is also anticipated, along with other investigational treatments like a functional cure for type 1 diabetes [11] - A 50% success rate in bringing products to market could significantly benefit the company, and clinical wins may positively impact stock performance [12]

Core Viewpoint - CRISPR Therapeutics AG is at the forefront of gene editing, having received approval for its first gene editing treatment, CASGEVY, which targets sickle cell disease and beta-thalassemia [2][4]. Company Overview - CRISPR Therapeutics' stock is currently trading at approximately $36.31, showing little change since 2018, and investors from 2020 and 2021 may be facing losses [3][11]. - The company has a significant cash reserve of approximately $1.86 billion as of March 31, 2025, which is expected to support future growth [8]. Product and Market Potential - CASGEVY has been approved in eight jurisdictions and has 65 authorized treatment centers globally, with expectations for significant patient initiation growth by 2025 [6]. - The total addressable market for CASGEVY is estimated to be around 60,000 patients, although this is small compared to larger markets for diseases like cancer and diabetes [6][7]. Future Outlook - The company anticipates that 2026 will mark a turning point, generating enough revenue from CASGEVY to support sustainable growth for ongoing and future clinical trials [8]. - Analysts have a 12-month price target for CRISPR Therapeutics stock at $71.75, indicating a potential upside of 97.60% from the current price [10][12]. Investment Sentiment - Despite the current challenges, over 69% of CRSP stock is owned by institutional investors, and buying has outpaced selling for the last nine quarters [11][12]. - The stock is rated as a Moderate Buy by analysts, although it is not currently considered a top investment compared to other stocks [12][14].