Summary of CRISPR Therapeutics FY Conference Call Company Overview - **Company**: CRISPR Therapeutics (NasdaqGM:CRSP) - **Event**: FY Conference Call on January 12, 2026 - **Focus**: Progress and updates on gene editing technologies and therapies Key Points Industry and Market Position - CRISPR Therapeutics is focused on transforming medicine through gene editing, aiming to develop cures for serious diseases [2][34] - The company has made significant advancements across four franchises, including hemoglobinopathies, in vivo gene editing, CAR-T therapies, and RNA-based therapies [2][3] Hemoglobinopathies - **Casgevy**: An approved product with multi-billion dollar revenue potential, showing a 3x increase in patient initiation and cell collections from 2024 to 2025 [5][6] - Revenue surpassed $100 million, with positive payer coverage in the U.S. and Europe [5][6] - Pediatric data for sickle cell and thalassemia shows promising results, expanding the addressable patient population [6][7] In Vivo Gene Editing - Focus on in vivo gene editing of the liver, with promising data for CTX310 targeting hypercholesterolemia [3][11] - A single infusion can lead to a nearly 50% reduction in LDL cholesterol and a 55% reduction in triglycerides [14][15] - The potential for a multi-billion dollar opportunity in treating cardiovascular diseases [17] CAR-T Therapies - CTX112 shows a 70% complete response rate in oncology settings, with a favorable safety profile [27][28] - The company is exploring maintenance therapies to enhance durability of responses [28] - In vivo CAR-T approaches are being developed, utilizing proprietary lipid nanoparticle systems [30] RNA-Based Therapies - CTX611 targets Factor XI, with a potential to revolutionize anticoagulation therapy, showing a 93% reduction in Factor XI antigen levels [21][23] - The siRNA market is projected to be a $20 billion opportunity, with CRISPR's approach aiming to reduce bleeding risks associated with current therapies [21][24] Regulatory and Future Outlook - The FDA is supportive of gene editing technologies, which may facilitate smoother regulatory pathways for CRISPR's programs [40][41] - The company anticipates pivotal trials for several programs, including A1AT and CTX310, in the coming years [36][39] - Continuous innovation and partnerships are expected to enhance the development of their pipeline [47][48] Financial Considerations - CRISPR aims to reduce healthcare costs through one-time gene editing solutions, potentially pricing therapies below $100,000 [50] - The company is focused on creating pharmacoeconomic benefits through its innovative therapies [50] Societal Impact and Diversity - The company acknowledges the importance of addressing racial and ethnic differences in treatment responses, particularly in hypertension medications [51] AI Integration - AI is being utilized to enhance efficiency in clinical trials and improve gene editing processes, particularly in protein and mRNA design [53] Conclusion - CRISPR Therapeutics is positioned for significant growth with a diverse pipeline of innovative therapies across multiple disease areas, supported by strong market potential and favorable regulatory conditions. The company is committed to continuous innovation and addressing healthcare affordability through its gene editing technologies [34][56]

CRISPR Corporate Update 44th Annual J.P. Morgan Healthcare Conference January 12, 2026 1 Forward-Looking Statements This presentation and related materials may contain statements regarding matters that are not historical facts and are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Suc ...

Core Viewpoint - The average brokerage recommendation (ABR) for CRISPR Therapeutics AG (CRSP) is 1.96, indicating a general suggestion to buy the stock, but reliance solely on this metric may not be advisable due to potential biases in brokerage recommendations [2][5][11]. Brokerage Recommendations - CRISPR Therapeutics has an ABR of 1.96, which is close to a "Strong Buy" rating, based on recommendations from 29 brokerage firms [2]. - Out of the 29 recommendations, 15 are classified as "Strong Buy" and 1 as "Buy," representing 51.7% and 3.5% of total recommendations, respectively [2]. Limitations of Brokerage Recommendations - Brokerage analysts often exhibit a strong positive bias in their ratings due to vested interests, leading to a higher number of "Strong Buy" recommendations compared to "Strong Sell" [6][11]. - Studies indicate that brokerage recommendations may not effectively guide investors toward stocks with the highest potential for price appreciation [5][11]. Zacks Rank Comparison - The Zacks Rank, a proprietary stock rating tool, categorizes stocks from 1 (Strong Buy) to 5 (Strong Sell) and is based on earnings estimate revisions, which are more reliable indicators of near-term stock performance [8][12]. - The Zacks Rank is updated more frequently than the ABR, reflecting timely changes in earnings estimates and business trends [13]. Current Earnings Estimates for CRISPR Therapeutics - The Zacks Consensus Estimate for CRISPR Therapeutics remains unchanged at -$6.34 for the current year, suggesting stable analyst views on the company's earnings prospects [14]. - Due to the unchanged consensus estimate and other factors, CRISPR Therapeutics holds a Zacks Rank of 3 (Hold), indicating a cautious approach despite the positive ABR [15].

Core Insights - CRISPR Therapeutics is set for a significant year in 2026, with multiple data releases and milestones anticipated across its portfolio [1][2] - The company is focusing on the acceleration of CASGEVY, which has multi-billion-dollar potential, alongside a diverse pipeline supported by advanced gene-editing technologies [1][2] Financial Position - The company starts 2026 with a robust balance sheet, holding approximately $2 billion in cash, cash equivalents, and marketable securities [1] Product Pipeline and Milestones - CASGEVY is expected to see continued global commercialization, with regulatory submissions for patients aged 5-11 anticipated in the first half of 2026 [4][8] - Updates on CTX310 and the Lp(a) program are expected in the second half of 2026, with CTX611's Phase 2 clinical data also anticipated during this period [4][8] - The company plans to initiate clinical trials for CTX460 in alpha-1 antitrypsin deficiency and CTX340 in refractory hypertension in mid-2026 and the first half of 2026, respectively [4][8] CASGEVY Overview - CASGEVY is a CRISPR/Cas9 gene-edited cell therapy approved for patients with sickle cell disease and transfusion-dependent beta thalassemia, showing significant revenue growth exceeding $100 million in 2025 [10][4] - The therapy has demonstrated a nearly three-fold increase in patient initiations and first cell collections in 2025 compared to 2024 [4] In Vivo and siRNA Programs - The company is advancing a diversified portfolio of in vivo gene editing programs utilizing its proprietary lipid nanoparticle (LNP) delivery platform [5][12] - The siRNA-based portfolio includes clinical-stage programs targeting cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics [6][17] Autoimmune Disease and Oncology - Zugocabtagene geleucel (zugo-cel) is progressing in clinical trials for autoimmune diseases and hematologic malignancies, with updates expected in the second half of 2026 [7][14] - The company has established collaborations to expand the development of zugo-cel in aggressive B-cell lymphomas [14][17]

Group 1 - In 2025, investors could have achieved 42% returns by investing in the top 10 performers of 2024, significantly outperforming the S&P 500's 16% gain [2] - Hindsight investing can lead to significant losses, as seen with Signature Bank and Ford Motor Co. in 2022, where they experienced declines of 63% and 42% respectively [3] - Current trends that drove growth in 2024 and 2025 are becoming less reliable, prompting a need for investors to adapt to new trends in the next 60 to 90 days [5] Group 2 - The anticipated trend of rate cuts in 2026 may be underestimated, with betting markets suggesting at least three cuts, which could benefit Rocket Cos. Inc. (RKT) [8][9] - Rocket Cos. is positioned to capitalize on potential refinancing activity if mortgage rates fall below 6%, following a recent upgrade to an "A" grade in the Stock Grader system [12][13] - Gene editing technologies are emerging as a significant trend, with Crispr Therapeutics AG (CRSP) being a leading company in this space, expected to see substantial revenue growth from its sickle-cell therapy [14][20] Group 3 - Evolv Technologies Holdings Inc. (EVLV) is positioned to benefit from increased demand for security solutions, particularly in public spaces, as it offers advanced weapon detection technology [22][24] - Evolv has shifted to a subscription model and improved its operations following a scandal, which may lead to better-than-expected growth in 2026 [25][26] - The overall market is showing signs of potential downturns, with historical parallels to previous market collapses, indicating that current optimism may be misplaced [27][28]

Core Insights - CRISPR Therapeutics will present at the 44th Annual J.P. Morgan Healthcare Conference on January 12, 2026, at 8:15 a.m. PT in San Francisco [1] Company Overview - CRISPR Therapeutics is a leading biopharmaceutical company focused on developing gene-based medicines for serious diseases, evolving from a research-stage organization to an industry leader [3] - The company achieved a historic milestone with the approval of CASGEVY (exa-cel), the world's first CRISPR-based therapy for sickle cell disease and transfusion-dependent beta thalassemia [3] - CRISPR Therapeutics has a diversified pipeline targeting hemoglobinopathies, oncology, regenerative medicine, cardiovascular and autoimmune diseases, and rare diseases [3] - The company is advancing its gene editing capabilities through the development of SyNTase™, a proprietary platform for precise and efficient gene correction [3] - Strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals, are established to enhance its impact [3] - The company is headquartered in Zug, Switzerland, with R&D operations in Boston, Massachusetts, and San Francisco, California [3]

Group 1 - The article promotes a weekly newsletter focused on stocks in the biotech, pharma, and healthcare industries, highlighting key trends and catalysts that influence market valuations [1] - Edmund Ingham, a biotech consultant with over 5 years of experience, leads the Haggerston BioHealth investing group, which caters to both novice and experienced investors [1] - The investing group provides insights on product sales, forecasts for major pharmaceutical companies, integrated financial statements, discounted cash flow analysis, and market-specific analyses [1]

Group 1 - The article promotes a weekly newsletter focused on stocks in the biotech, pharma, and healthcare industries, highlighting key trends and catalysts that influence market valuations [1] - Edmund Ingham, a biotech consultant with over 5 years of experience, leads the Haggerston BioHealth investing group, which caters to both novice and experienced investors [1] - The investing group provides insights such as buy and sell ratings, product sales forecasts for major pharmaceutical companies, and detailed financial analyses [1]

Core Viewpoint - The current rotation within ARK Invest indicates a strong belief that 2026 will be a pivotal year for the genomic revolution [1] Group 1: Investment Strategy - ARK Invest is significantly reducing its holdings in legacy winners like Tesla and Rocket Lab to invest millions in gene-editing companies [2] - The firm is focusing on acquiring shares of CRISPR Therapeutics, Intellia Therapeutics, Beam Therapeutics, and Pacific Biosciences as 2025 ends [3] Group 2: Thematic Focus - Cathie Wood's investment thesis combines artificial intelligence with programmable biology, emphasizing AI's transformative potential in healthcare [4] - The multiomics flywheel framework suggests that AI enhances all aspects of multiomics, from biological data generation to disease diagnosis and drug development [4] Group 3: Technological Advancements - Improved algorithms are making gene sequencing faster, while cheaper sequencing provides more data for AI learning, leading to breakthroughs in gene editing and new treatment possibilities [5] Group 4: Holdings Overview - By late December 2025, ARK increased its stake in CRISPR Therapeutics to over 5% of the ARKK Innovation Fund, with Beam Therapeutics at 3.41%, Twist at 2%, and Intellia at 1.14% [6] - ARK also manages the Ark Genomic Revolution Fund, which invests in companies across healthcare and information technology relevant to the genomics theme [6]

Core Insights - Artificial intelligence (AI) is identified as the greatest investment opportunity of the current era, with a strong emphasis on the urgent need for energy to support its growth [1][2][3] Group 1: AI and Energy Demand - AI technologies, particularly large language models like ChatGPT, are extremely energy-intensive, with data centers consuming as much energy as small cities [2] - The increasing demand for AI is straining global power grids, leading to rising electricity prices and a need for utilities to expand capacity [2] - Industry leaders, including Sam Altman and Elon Musk, have highlighted the critical link between AI's future and energy availability, with Musk warning of potential electricity shortages as early as next year [2] Group 2: Investment Opportunity - A specific company is positioned as a key player in the AI energy sector, owning critical energy infrastructure assets that will benefit from the anticipated surge in energy demand from AI data centers [3][7] - This company is not a chipmaker or cloud platform but is described as a "toll booth" operator in the energy market, profiting from the export of American LNG and the onshoring of manufacturing due to tariffs [5][6] - The company is debt-free and has significant cash reserves, equating to nearly one-third of its market capitalization, making it an attractive investment option [8] Group 3: Market Position and Valuation - The company is involved in large-scale engineering, procurement, and construction projects across various energy sectors, including nuclear energy, which is crucial for America's future power strategy [7] - It is trading at a low valuation of less than 7 times earnings, excluding cash and investments, indicating a potential for significant upside [10] - The company also holds a substantial equity stake in another AI-related venture, providing investors with indirect exposure to multiple growth opportunities in the AI sector [9] Group 4: Future Outlook - The ongoing AI infrastructure supercycle, combined with the onshoring boom and a surge in U.S. LNG exports, positions this company favorably for future growth [14] - The influx of talent into the AI sector ensures continuous innovation and advancements, further solidifying the importance of investing in AI-related companies [12] - The overall message emphasizes the urgency for investors to act now to capitalize on the potential for substantial returns in the AI and energy markets [15][19]