Sana Biotechnology (NasdaqGS:SANA) FY Conference Transcript

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.