Core Viewpoint - Prime Medicine's technology, Prime Editing, is positioned as a transformative gene editing tool with the potential to significantly impact patient lives by enabling a wide range of genetic modifications [3]. Company Overview - Prime Medicine is focused on advancing gene editing technology, specifically through Prime Editing, which is described as the most versatile method for genome manipulation [3]. - The company believes that gene editing will play a crucial role in addressing human diseases both now and in the future [3]. Technology Insights - Prime Editing can perform all functions of existing gene editing technologies while also allowing for additional types of edits, including large incisions, large insertions, hotspot editing, missense mutations, and transversion mutations [3]. - The technology offers extensive possibilities for treating various diseases, highlighting its potential to revolutionize the field of genetic medicine [3].

Summary of Prime Medicine FY Conference Call Company Overview - **Company**: Prime Medicine (NasdaqGM:PRME) - **Industry**: Biotechnology, specifically focused on gene editing technologies Core Technology - **Prime Editing**: Described as the most versatile and safest gene editing technology, capable of performing various types of edits including large deletions, insertions, and specific mutations [2][3] - **Intellectual Property**: Strong IP position with exclusive licenses from the Broad Institute, covering foundational aspects of Prime Editing [4][12] Clinical Programs - **Liver Franchise**: Focus on two main programs: - **Wilson Disease**: IND submission planned for the first half of 2026, with proof of concept data expected in 2027. Estimated patient population in the U.S. and EU is around 10,000, with potential for 25,000 patients targeted with six common mutations [5][12][13] - **Alpha-1 Antitrypsin Deficiency**: IND submission planned for mid-2026, with similar proof of concept data expected in 2027. Estimated patient population is 20,000-30,000 in the U.S. and Europe [5][14] - **Cystic Fibrosis**: Ongoing progress with plans to share proof of concept data in 2026, targeting a significant unmet need in patients who cannot tolerate current treatments [5][6] Business Development Strategy - **Collaboration with BMS**: Focus on ex vivo CAR T therapies for oncology, hematology, and immunology, with a $110 million upfront payment and significant preclinical milestones [8] - **Expansion Plans**: Emphasis on business development to broaden the reach of Prime Editing technology, including potential applications in neuro diseases and other cell therapies [20][22] Regulatory Environment - **Regulatory Changes**: Anticipated improvements in the regulatory framework that may allow for faster and cheaper movement of therapies into clinical trials [3][10] - **IND and CTA Submissions**: Clarification that both submissions are being pursued for global studies, with flexibility on which may occur first [27][28] Financial Position - **Cash Reserves**: Approximately $227 million available, with key milestones including the initiation of clinical trials for the liver franchise programs and potential arbitration resolution with Beam [47][48] Key Milestones and Future Outlook - **2026 Goals**: Focus on getting the first two in vivo programs into the clinic and achieving proof of concept data by 2027 [20][46] - **Market Potential**: High-value programs identified as those with a high probability of success and large addressable markets, particularly in orphan diseases [6][7] Risks and Challenges - **Market Competition**: Acknowledgment of competition in the gene editing space, particularly for alpha-1 antitrypsin deficiency, but confidence in Prime Editing's superior approach [39][41] - **Technological Responsibility**: Emphasis on the importance of making the right decisions to ensure the technology reaches patients effectively [42] Conclusion - **Strategic Focus**: Prime Medicine is concentrating on its liver franchise and cystic fibrosis programs, with a clear path towards clinical trials and proof of concept data, while also exploring business development opportunities to expand its technology's reach [20][46]

Delivering on the promise of Prime Editing Corporate Presentation January 2026 1 Forward Looking Statements This presentation contains forward-looking statements of Prime Medicine, Inc. ("Prime", "we" or "our") within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements contain information about our current and future prospects and our operations, which are based on currently available information. All statements other than statements of historica ...

Summary of Prime Medicine FY Conference Call Company Overview - **Company**: Prime Medicine (NasdaqGM:PRME) - **Focus**: Gene editing technology, specifically prime editing Key Points on Prime Editing Technology - **Definition**: Prime editing is described as the most versatile and safest method for genome editing, allowing for precise and permanent corrections in the DNA sequence [6][14] - **Comparison with Other Technologies**: - **CRISPR**: Effective for knocking out genes but has limitations such as off-target effects and potential immunogenicity [6][7][9] - **Base Editing**: Allows for single-letter changes in DNA but is limited to specific types of mutations [9][10][14] - **Prime Editing Advantages**: Capable of correcting larger mutations and multiple mutations simultaneously, with lower off-target effects [14][15] Industry Context - **Competitive Landscape**: Other companies, such as Tessera and Regeneron, are also developing prime editing technologies, indicating a competitive market [34] - **Market Need**: There is a significant unmet need for therapies targeting conditions like Wilson disease and alpha-1 antitrypsin deficiency, which are suitable for prime editing [32][33] Corporate Strategy and Pipeline - **Recent Changes**: The company underwent restructuring to focus on high-value areas, reducing the number of programs from 18 to prioritize those with higher probabilities of success [31][32] - **Key Programs**: - **Wilson Disease**: Targeting specific mutations with no existing gene editing therapies [32] - **Alpha-1 Antitrypsin Deficiency**: A competitive area where prime editing is believed to provide superior outcomes [33] - **Cystic Fibrosis and Ex Vivo CAR T Programs**: Continued focus on these areas, while deprioritizing ocular and neurological programs [38][40] Development and Clinical Trials - **IND Timeline**: The company aims to submit an Investigational New Drug (IND) application for Wilson disease in the first half of 2026, with proof of concept data expected by 2027 [48][49] - **Trial Design**: The Wilson disease trial will be a dose escalation study focusing on safety and efficacy measures, targeting patients with specific mutations [52][54] - **Biomarkers**: Various biomarkers will be used to assess treatment efficacy, including copper PET studies and ceruloplasmin levels [54] Off-Target Editing and Safety - **Off-Target Analysis**: Prime Medicine has conducted extensive off-target analysis for its programs, reporting no evidence of off-target effects in their first program for chronic granulomatous disease [16][31] - **Assay Development**: The company has developed its own assays for measuring off-target effects, which may differ from those used by competitors [17][30] Future Outlook - **Partnerships**: While the company is open to partnerships, it sees significant value in independently advancing its key programs [41][42] - **Long-Term Vision**: Prime Medicine aims to expand its portfolio beyond single products and diseases, leveraging its technology for a broader range of genetic conditions [41] Conclusion - Prime Medicine is positioned as a leader in the gene editing space with its prime editing technology, focusing on high-value therapeutic areas with significant unmet needs. The company is preparing for upcoming clinical trials and aims to establish itself as a key player in the competitive landscape of gene editing therapies.

Core Insights - Prime Medicine, Inc. has appointed Matthew Hawryluk, Ph.D., M.B.A. as Chief Business Officer to lead corporate and business development initiatives, corporate strategy, and alliance management [1][2][4] - The company aims to expand the reach of its Prime Editing technology through strategic partnerships and collaborations, which are considered a core pillar of its strategy [2][4] - Dr. Hawryluk brings nearly two decades of experience in the biotechnology industry, having previously held leadership roles at AIRNA Corporation and Gritstone bio, Inc., where he led significant financing and collaborations [2][3] Company Overview - Prime Medicine is focused on delivering a new class of one-time curative genetic therapies using its proprietary Prime Editing platform, which allows precise and efficient gene editing [5][6] - The company is advancing a diversified portfolio of investigational therapeutic programs targeting liver, lung, immunology, and oncology diseases, with plans to expand into additional genetic and immunological diseases, cancers, and infectious diseases [6][7] - Prime Editing technology has the potential to repair various genetic mutations and is designed to minimize unwanted DNA modifications, unlocking opportunities across thousands of potential indications [5][6]

Company Overview - Rubrik is valued at $15.2 billion and specializes in data security, backup, recovery, and cloud-based protection, particularly relevant in the context of increasing ransomware and data breach risks [3] - The company reported a significant increase in subscription annual recurring revenue (ARR) to over $1.25 billion, up 36% year-over-year, with cloud ARR surging 57% to $1.1 billion [2] Financial Performance - Overall revenue increased by 51% to $310 million, with subscription revenue rising by 55% [1] - Adjusted net loss narrowed to $0.03 from $0.40 in the same quarter last year, and adjusted gross margin improved to 82% from 77% [1] - Free cash flow improved to $57.5 million from a negative $32 million last year, indicating a stronger financial position [6] Market Position and Analyst Sentiment - Analysts predict Rubrik's revenue to grow by 39.2% in 2025 and 25% in 2026, reflecting confidence in the company's growth trajectory [7] - The stock is rated as a "Strong Buy" by 18 out of 21 analysts, with an average target price of $114.95, suggesting a potential upside of 49.8% [8] Investment Activity - Cathie Wood's ARK Next Generation Internet ETF purchased 66,836 shares of Rubrik for $6.6 million, increasing total investment in the company to $35 million [3][4] - Wood's investment strategy focuses on disruptive innovation and long-term growth, aligning with Rubrik's fundamentals and market trends [5][7]

Core Insights - Prime Medicine has secured up to $24 million in additional funding from the Cystic Fibrosis Foundation to accelerate the development of Prime Editors for cystic fibrosis (CF) [1][2][3] - The funding aims to enhance Prime Editing technology, which has the potential to correct a wide range of genetic mutations affecting over 93% of individuals with CF [1][2] - The initial focus will be on the G542X mutation, a prevalent CF-causing mutation with no current therapies available [2] Company Overview - Prime Medicine is a biotechnology company dedicated to developing one-time curative genetic therapies using its proprietary Prime Editing platform, which allows precise and efficient gene editing [5][6] - The company is advancing a diversified portfolio of therapeutic programs targeting liver, lung, immunology, and oncology diseases [6][7] - Prime Editing technology is designed to minimize unwanted DNA modifications while effectively repairing various genetic mutations across different tissues and organs [5] Funding Details - The CF Foundation's investment builds on an earlier agreement from January 2024 and will be provided in two tranches, with the first tranche including a $6 million equity investment [2][3] - The funding is contingent upon meeting certain scientific milestones and closing conditions [3] Disease Context - Cystic fibrosis is a serious genetic disease caused by mutations in the CFTR gene, affecting approximately 100,000 people globally, including over 40,000 in the United States [4] - Current disease-modifying therapies are not curative and may be ineffective for certain mutations, highlighting the need for innovative treatments [4]