Summary of Sana Biotechnology FY Conference Call Company Overview - **Company**: Sana Biotechnology (NasdaqGS:SANA) - **Event**: 44th Annual J.P. Morgan Healthcare Conference - **Date**: January 14, 2026 - **Presenter**: Steve Harr, President and CEO Core Industry Focus - **Industry**: Biotechnology, specifically in cell therapy and gene editing - **Target Disease**: Type 1 diabetes and potential applications in blood cancers and autoimmune diseases Key Points and Arguments Goals and Progress - **Main Goals**: - Overcome allogeneic rejection in cell therapy to make it universally available - Develop effective delivery methods for genetic payloads to cells [2][3] - **Focus Area**: Type 1 diabetes, a significant unmet medical need with high demand for better treatment options [3][9] Achievements - **Cell Transplantation**: Successfully demonstrated the ability to transplant cells that evade the immune system [4] - **Master Cell Bank**: Established a master cell bank for consistent production of pancreatic beta cells [4][28] - **Regulatory Engagement**: Engaged with global regulators to align on future plans, including filing an Investigational New Drug (IND) application [4][29] Clinical Development - **Phase One Study**: Plans to file IND and begin Phase One study within the year, aiming for quick proof of concept regarding immune evasion and functional insulin production [4][30] - **Patient Outcomes**: A patient who received the therapy has shown no adverse events and continued insulin production for over a year [16][21] Scientific Insights - **Mechanism of Action**: - Knocking out MHC class I and II to prevent immune rejection, combined with overexpression of CD47 to evade both adaptive and innate immunity [13][14] - Evidence of survival and function of transplanted cells through C-peptide levels and PET MRI scans [17][21] Market Potential - **Unmet Need**: Curing type 1 diabetes could equate to curing both HIV and multiple sclerosis in the U.S., highlighting the vast market potential [9] - **Scalability**: The goal is to create a scalable therapy that can treat millions of patients, with a focus on manufacturing efficiency [28][58] Future Directions - **In Vivo CAR T Cells**: Development of a best-in-class in vivo CAR T platform, with plans to initiate trials in cancer and autoimmune diseases [43][60] - **Partnership Strategy**: Considering partnerships to accelerate development in competitive areas like B cell cancers and autoimmune diseases [60] Regulatory and Manufacturing Challenges - **Regulatory Complexity**: Navigating the complexities of novel immunology, gene editing, and stem cell biology with regulators [51] - **Manufacturing Scale**: Challenges in scaling production from Phase One to commercial levels, focusing on maintaining genomic stability and purity [45][47] Investment and Financial Outlook - **Investment Needs**: Significant investment required for manufacturing scale-up and clinical development, with ongoing financial commitments expected [56][58] Additional Important Insights - **Patient Population for Trials**: Initial trials will focus on adults with type 1 diabetes, with plans to expand to younger populations as data is gathered [52][53] - **Global Reach**: The Phase One study will not be limited to the U.S. but will include other geographies [54] This summary encapsulates the key points discussed during the conference call, highlighting Sana Biotechnology's strategic focus, achievements, and future plans in the biotechnology sector.