Data Demonstrate Potent in vivo Gene Editing of Hematopoietic Stem Cells (HSCs) in the Bone Marrow with Systemic Delivery in Preclinical Murine Models Using Fusogen Technology Broadens Application of Fusogen Technology Beyond T Cells to Second Cell Type, HSCs, Showing Potent and Specific in vivo Delivery Underscores Ability of Fusogen Technology to Deliver Diverse Payloads, including CRISPR Gene-Editing and Base-Editing Machinery Highlights Potential of Fusogen Platform in Diseases like Sickle Cell Disease ...

Question-and-Answer SessionSo Steve, why don't you kick it off? And I think maybe just remind us about the platform, but also remind us about how Sana is playing in vivo CAR-T because it's fashionable and everyone is playing in there, but I feel like you guys are not necessarily just starting up in there, for example.Steven HarrPresident, CEO & Director Yes. First of all, thank you, everybody, for joining. Thank you to Evercore for having us. I'm sure you guys recognize, we may make a few forward-looking st ...

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.

Question-and-Answer SessionWell, so why don't we just start off with a little bit of an overview on Sana. I know you recently streamlined our pipeline to focus on SC451, which is your T1D islet cell product. And also SG293, which is your in vivo CAR T product. Just talk a little bit about that decision and what Sana looks like going forward?Steven HarrPresident, CEO & Director Sure. First of all, thanks, everybody, for joining us, both in the room and online. And I think you guys know we'll be making forwar ...

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]

Sana Biotechnology (NasdaqGS:SANA) FY 2025 Conference December 02, 2025 02:30 PM ET Company ParticipantsSteve Harr - CEOConference Call ParticipantsNone - Analyst 2None - AnalystNone - Analyst 1NoneYour biotech analyst here at Citi, and today it's my pleasure to be hosting Sana Biotechnology for a fireside chat at Citi's Global Healthcare Conference. I'm joined today by CEO Steve Harr. Steve, thank you so much for being here today.Steve HarrThank you for having me, Samantha.So why don't we just start off wi ...

Sana Biotechnology (NasdaqGS:SANA) FY 2025 Conference December 02, 2025 02:30 PM ET Speaker4your biotech analyst here at Citi, and today it's my pleasure to be hosting Sana Biotechnology for a fireside chat at Citi's Global Healthcare Conference. I'm joined today by CEO Steve Harr. Steve, thank you so much for being here today.Speaker0Thank you for having me, Samantha.So why don't we just start off with a little bit of an overview on Sana? I know you recently streamlined your pipeline to focus on SC451, whi ...

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]