Summary of Sana Biotechnology FY Conference Call (December 03, 2025) Company Overview - **Company**: Sana Biotechnology (NasdaqGS:SANA) - **Focus**: Development of cell therapies, particularly in vivo CAR T and treatments for Type 1 diabetes Key Points Industry Context - The field of cell therapy, especially in vivo CAR T, is gaining traction with multiple companies entering the space [1][2] - Type 1 diabetes affects approximately 9 million people and is projected to grow to about 15 billion by 2040 [3] Core Product: SC451 - **Objective**: Aiming for a functional cure for Type 1 diabetes through gene-modified islet transplantation [2][6] - **Mechanism**: Gene-modified islets can function without immunosuppression, a significant advancement over traditional methods [5][6] - **Current Status**: IND (Investigational New Drug) application expected in 2026, with hopes to begin clinical studies [6][17] Clinical Insights - Previous studies have shown that gene-modified islets can survive and function without the need for insulin [6][8] - The goal is to achieve normal blood sugar levels and eliminate the need for insulin in patients [15][16] - Current data indicates that a dose of around 1 billion cells may be necessary for achieving euglycemia [15] Manufacturing and Regulatory Pathway - The company is in discussions with global regulators, including the FDA, to ensure compliance for human testing [16][23] - Challenges include ensuring genomic stability and product purity during the manufacturing process [20][21] - The company has made significant progress in developing a master cell bank for consistent product quality [22][23] Competitive Landscape - Other companies, such as Vertex, have reported success with stem cell-derived islet transplants, but often require immunosuppression [12][13] - Sana's approach aims to overcome the need for immunosuppression, potentially offering a more scalable solution [5][6] In Vivo CAR T Development - The in vivo CAR T program focuses on delivering a DNA plasmid specifically to T cells, enhancing manufacturability and safety [31][32] - The company has demonstrated effective B cell depletion in non-human primates, indicating potential for human application [34] Future Outlook - The company is optimistic about achieving breakthrough designation based on upcoming data [27][28] - Phase 1 trials are anticipated to involve 12-15 patients, with preparations underway for manufacturing [29] Additional Considerations - The company acknowledges potential challenges in scaling manufacturing and ensuring consistent cell viability post-transplant [19][24] - T cell fitness and individual patient immune responses may impact the effectiveness of CAR T therapies [36] Conclusion Sana Biotechnology is positioned at the forefront of innovative cell therapies, particularly for Type 1 diabetes and in vivo CAR T applications. The company is making strides in overcoming traditional challenges associated with immunosuppression and manufacturing, with a clear path toward clinical trials and potential market entry.

Overview - The company has streamlined its pipeline to focus on two key products: SC451, a T1D islet cell product, and SG293, an in vivo CAR T product [1] Group 1: Type 1 Diabetes (T1D) - Type 1 diabetes is a well-understood disease characterized by the immune system attacking pancreatic beta cells, which are responsible for insulin production [1] - Historically, prior to insulin therapy, T1D was a fatal condition leading to rapid death within months [1]

Financial Data and Key Metrics Changes - The company has streamlined its pipeline to focus on SC451 for type 1 diabetes and SG293 for in vivo CAR-T therapy, indicating a strategic shift towards high-potential products [2][6] - The company aims to initiate a clinical study for SC451 next year, with an IND submission expected soon, reflecting confidence in its development timeline [21][56] Business Line Data and Key Metrics Changes - The type 1 diabetes program is positioned as a one-time curative therapy, with the goal of eliminating the need for insulin and immunosuppression for patients [6][56] - The in vivo CAR-T platform is being developed to target various blood cancers and autoimmune disorders, with a focus on cell specificity and safety [8][46] Market Data and Key Metrics Changes - There are approximately 10 million people with type 1 diabetes in the U.S., projected to grow to 15 million in 15 years, highlighting a significant market opportunity [4][23] - The company is aware of the competitive landscape in the CAR-T space but believes its differentiated approach could lead to a best-in-class product [9][45] Company Strategy and Development Direction - The primary focus remains on advancing the type 1 diabetes program while exploring partnerships to enhance financial resilience and success probability [29][31] - The company is also considering partnerships for the in vivo CAR-T platform to accelerate development and leverage external expertise [49][50] Management's Comments on Operating Environment and Future Outlook - Management emphasizes the high unmet need in type 1 diabetes and the urgency to deliver a safe and effective therapy [44][56] - The company is optimistic about the regulatory path and believes it can navigate the complexities of manufacturing and clinical trials effectively [15][24] Other Important Information - The company has established a master cell bank for its gene-modified pluripotent stem cells, which is crucial for consistent product manufacturing [20][19] - Safety remains a primary concern, with management focusing on mitigating risks associated with severe hypoglycemia and potential tumor formation [14][18] Q&A Session Summary Question: What is the current status of the IND preparation for type 1 diabetes? - The company is engaged in dialogues with regulators and is working on completing the non-clinical toxicology package and GMP manufacturing to move forward with human testing [11][15] Question: How does the company plan to scale manufacturing for the type 1 diabetes therapy? - The company acknowledges the challenges of scaling but believes it can solve the manufacturing issues internally while also exploring partnerships for scientific support [33][29] Question: What are the key differentiators of the in vivo CAR-T platform? - The in vivo CAR-T platform utilizes virus-like particles for targeted delivery, which allows for cell-specific treatment without the complications associated with traditional methods [46][47] Question: How does the company view potential partnerships for its programs? - The company is open to partnerships that can enhance its financial stability and improve the probability of success, particularly for the in vivo CAR-T platform [29][49] Question: What are the anticipated timelines for clinical trials? - The company aims to initiate a phase 1 trial for the type 1 diabetes therapy next year, with data expected to emerge quickly thereafter [21][56]

Financial Data and Key Metrics Changes - The company has streamlined its pipeline to focus on SC451 for type 1 diabetes and SG293 for in vivo CAR T therapy, indicating a strategic shift towards high-potential products [2][6] - The company aims to initiate a clinical study for SC451 next year, with an IND submission expected soon, reflecting confidence in its development timeline [6][21] Business Line Data and Key Metrics Changes - The type 1 diabetes program is positioned as a one-time curative therapy, with the goal of eliminating the need for insulin and immunosuppression for patients [6][56] - The in vivo CAR T platform is designed to target various blood cancers and autoimmune disorders, with a focus on cell specificity and safety [8][45] Market Data and Key Metrics Changes - There are approximately 10 million people with type 1 diabetes in the U.S., projected to grow to 15 million in 15 years, highlighting a significant market opportunity [4][23] - The company is aware of the competitive landscape in the CAR T space but believes its differentiated approach could lead to a best-in-class product [9][45] Company Strategy and Development Direction - The primary focus remains on advancing the type 1 diabetes program while exploring partnerships for the in vivo CAR T platform to enhance financial resilience and success probability [29][56] - The company is committed to ensuring safety and efficacy in its therapies, with rigorous testing and regulatory alignment as key priorities [11][15] Management's Comments on Operating Environment and Future Outlook - Management emphasizes the high unmet need in type 1 diabetes and the urgency to deliver a safe and effective therapy [44][56] - The company is optimistic about its clinical path and believes it can achieve significant milestones in the near term, including entering the clinic next year [55][57] Other Important Information - The company has developed a master cell bank that retains pluripotency and does not mutate, which is crucial for the safety and efficacy of its therapies [20][13] - There is a focus on ensuring product purity to prevent potential long-term safety issues, such as tumor formation [18][15] Q&A Session Summary Question: What is the current status of the IND preparation for type 1 diabetes? - The company is engaged in dialogues with regulators and is working on completing the non-clinical toxicology package and GMP manufacturing to move forward with human testing [11][19] Question: How does the company plan to scale manufacturing for its therapies? - The company acknowledges that scaling manufacturing is a significant challenge and is focused on ensuring consistent delivery at a scale that meets patient needs [22][24] Question: What are the company's thoughts on potential partnerships for type 1 diabetes? - The company is open to partnerships that can enhance financial resilience and improve the probability of success, but it maintains a high bar for any potential deal [28][29] Question: How does the company differentiate its in vivo CAR T platform from competitors? - The company utilizes a unique virus-like particle approach for targeted delivery, which allows for cell-specific treatment without the complications associated with other methods [46][45] Question: What are the anticipated timelines for clinical trials and data release? - The company aims to initiate a phase I trial for type 1 diabetes next year, with data expected to be available shortly after [21][55]

Financial Data and Key Metrics Changes - The company has streamlined its pipeline to focus on SC451 for type 1 diabetes and SG293 for in vivo CAR-T, indicating a strategic shift towards more targeted therapies [2][6] - The company aims to initiate a clinical study for SC451 next year, with an IND submission expected soon, reflecting confidence in its development timeline [6][21] Business Line Data and Key Metrics Changes - The type 1 diabetes program is positioned as a one-time curative therapy, with the potential to significantly improve patient outcomes by eliminating the need for insulin and immunosuppression [6][12] - The in vivo CAR-T platform is designed to deliver treatments for various blood cancers and autoimmune disorders, with a focus on cell specificity and safety [8][42] Market Data and Key Metrics Changes - There are approximately 10 million people with type 1 diabetes in the U.S., projected to grow to 15 million in 15 years, highlighting a significant market opportunity [4][23] - The company is preparing to address a large unmet need in the type 1 diabetes market, emphasizing the urgency and potential impact of its therapies [41][52] Company Strategy and Development Direction - The primary focus remains on advancing the type 1 diabetes program, with the in vivo CAR-T platform serving as a secondary opportunity for diversification and potential capital generation [53][54] - The company is actively engaging with global regulators to ensure alignment on clinical protocols, aiming for a straightforward path to human testing [15][40] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the type 1 diabetes program, citing successful gene edits and the establishment of a master cell bank as significant de-risking events [52][54] - The company acknowledges the challenges of scaling manufacturing to meet potential demand but believes it can address these issues independently [30][31] Other Important Information - The company has established a master cell bank that retains pluripotency and does not mutate, which is crucial for the safety and efficacy of its therapies [20][21] - There is a recognition of the need for partnerships to enhance financial resiliency and improve the probability of success for its therapies, particularly in the context of type 1 diabetes [28][29] Q&A Session Summary Question: What is the current status of the IND preparation for type 1 diabetes? - The company is focused on completing the non-clinical toxicology package and GMP manufacturing to move forward with human testing [11][19] Question: How does the company plan to scale manufacturing for the type 1 diabetes therapy? - The company is addressing both the science and capital aspects of scaling, emphasizing the need for a partner to help with scientific challenges [28][29] Question: What are the key safety concerns associated with the type 1 diabetes therapy? - Management highlighted the risks of severe hypoglycemia and potential tumor formation as primary safety concerns that need to be managed during preclinical testing [12][14]

Core Insights - Sana Biotechnology, Inc. (NASDAQ:SANA) experienced a significant stock increase of 14.53% to $4.02 as investors anticipated upcoming business updates [1][4]. Company Overview - Sana Biotechnology is focused on developing engineered cells as medicines [5]. Financial Performance - In Q3, the company reduced its net loss by 30% to $42.15 million from $59.92 million year-on-year [6]. - Total operating expenses decreased by 29.5% to $43.51 million compared to $61.76 million in the same period last year [7]. Upcoming Events - The CEO, Steve Harr, will present a business overview at the Citi 2025 Global Healthcare Conference on December 2 and the Evercore Healthcare Conference on December 3 [2]. - Investors are particularly interested in updates regarding drug candidates SC451 and SG293, which target diabetes and B-Cell cancer/autoimmune diseases, respectively [3].

Core Insights - Sana Biotechnology, Inc. is set to present at two investor conferences in December, providing a business overview and updates from CEO Steve Harr [1][5] - The presentations will be accessible via webcast on the company's Investor Relations page, with replays available for 30 days post-conference [2] Company Overview - Sana Biotechnology focuses on creating engineered cells as medicines, aiming to repair and control genes, and replace missing or damaged cells [3] - The company operates in Seattle, WA, Cambridge, MA, and South San Francisco, CA, with a vision to make therapies broadly available to patients [3]

Industry Overview - Genomics is a comprehensive study of genomes, focusing on the complete set of DNA in an organism, and is gaining interest from pharmaceutical and biotechnology companies due to recent breakthroughs [1] - The distinction between genetics and genomics is important; genetics focuses on individual genes, while genomics aims to characterize all genes and their interactions within a living system [2] - Insights from genomics are increasingly used to develop targeted treatments and advance personalized medicine, addressing the rising demand for innovative medical therapies [3] Technological Advancements - The field of genomics has led to the emergence of synthetic biology, which applies engineering principles to biology for applications such as drug discovery and gene editing [4] - Significant progress in genomics has been driven by reductions in the cost, time, and effort required for genome sequencing, with Illumina being a major player in this space [5] - Genome-editing technologies, particularly CRISPR/Cas9, are being explored by companies like CRISPR Therapeutics AG and Intellia Therapeutics to treat diseases caused by genetic mutations [7] Market Projections - The genomics market is projected to reach $80.17 billion by 2032, while the synthetic biology market was valued at $16.2 billion in 2024 and is expected to grow at a CAGR of 17.30% from 2025 to 2030 [8] Investment Opportunities - Companies such as Editas Medicine, Sana Biotechnology, and Pacific Biosciences of California are highlighted as potential investment opportunities in the genomics and synthetic biology sectors [9] - Editas Medicine is developing EDIT-401, a gene editing therapy aimed at reducing LDL-cholesterol, showing over 90% reduction in preclinical tests [10][11] - Sana Biotechnology is focusing on cell engineering platforms for diseases like type 1 diabetes and B-cell cancers, with plans for IND applications in 2026 and 2027 [15][16][17] - Pacific Biosciences of California specializes in advanced sequencing solutions, with a focus on HiFi long-read sequencing technology, and has seen a 53.9% increase in shares over the past six months [18][19]

Core Viewpoint - Analysts are optimistic about Sana Biotechnology Inc. (NASDAQ: SANA), with multiple Buy ratings and a focus on its strategic initiatives and financial stability, suggesting it is a strong investment opportunity. Group 1: Analyst Ratings and Price Targets - Bank of America Securities analyst Alec Stranahan reaffirmed a Buy rating on Sana Biotechnology and set a price target of $6.00 [1] - TD Cowen analyst Marc Frahm also maintained a Buy rating, highlighting the company's strategic focus on the SC451 program while pausing allogeneic CAR T development to conserve cash [2] - Citizens initiated coverage of Sana Biotechnology with a Market Perform rating, indicating a positive outlook for the company [4] Group 2: Financial Position and Strategic Focus - Sana Biotechnology has a cash position of $170 million, which supports its strategic focus and financial stability [3] - The company is advancing its SG293 program, with an Investigational New Drug (IND) filing possible by 2027, and has gained FDA clarity on key steps toward an IND filing for SC451 in 2026 [3] - As of August, the company had $143 million in cash, positioning it well to enhance its diabetes program [5] Group 3: Market Potential and Growth Projections - Eric Jackson from EMJ Capital believes Sana Biotechnology could become a $100 billion company, driven by its diabetes therapy targeting 10% of the global Type 1 diabetes population at $100,000 per patient [5] - The company develops engineered cells as medicines to treat various diseases, focusing on gene repair, damaged cell replacement, and conditions like cancer and diabetes [6]

Summary of Sana Biotechnology Conference Call Company Overview - **Company**: Sana Biotechnology (NasdaqGS:SANA) - **Focus**: Development of gene-modified therapies, particularly for diabetes and CAR T cell therapies Key Points Industry Context - The diabetes market has approximately **10 million patients** and is growing at a **mid-single digit percentage** annually, with no significant innovations in therapy for about **100 years** [4][5] - There is a high level of patient dissatisfaction due to the burdens of managing blood glucose and the severe complications associated with diabetes [5] Core Programs - **SC451**: A gene-modified stem cell-derived islet aimed at treating type 1 diabetes, with the goal of achieving a functional cure [3][4] - The treatment aims to eliminate the need for insulin shots and monitoring, and avoid immunosuppression [3] - The company plans to begin a study for SC451 next year, indicating confidence in the program's progress [4] - **In Vivo Delivery Platform**: Focused on CAR T cell therapies, with promising data suggesting best-in-class safety and efficacy [6][34] Scientific Innovations - The company has developed a method to overcome both allogeneic and autoimmune rejection through specific gene modifications, including knocking out MHC class I and II and overexpressing CD47 [10][11] - A proof-of-concept trial in Uppsala demonstrated that gene-modified islets can survive and function in a type 1 diabetes patient without immunologic rejection [12][13] Regulatory and Developmental Milestones - The company is in discussions with the FDA regarding the IND application for SC451, with two main tasks remaining: finalizing the manufacturing process and completing the non-clinical pharmacotoxicology package [21][22] - The timeline for the IND filing is projected for **2026**, with potential delays depending on the tech transfer and GLP tox study outcomes [25][26] Clinical Trial Design - The Phase I trial design is expected to involve a modestly larger patient population than previous studies due to the broader patient demographic and the absence of immunosuppression [30][31] Financial Considerations - The company emphasizes the importance of not compromising its diabetes program while exploring funding options for the In Vivo CAR T program, including potential partnerships or direct investments [40] Future Outlook - The company is optimistic about the scalability of its gene-modified therapies and aims to progress towards a registration study and commercial launch [28][29] - There is a commitment to maintaining focus on diabetes due to its significant market potential and the advancements made in the underlying science [40][42] Additional Insights - The company acknowledges the inherent risks in translating animal data to human applications but expresses confidence based on extensive preclinical testing [19][20] - The CEO highlighted the importance of specificity and self-replicating capabilities in their CAR T cell therapies, which could position them favorably in the competitive landscape [38][39]