Sana Biotechnology (SANA) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Strategic vision and technology focus

• Aims to transform medicine by enabling gene modification and cell-based therapies, focusing on overcoming allogeneic transplant rejection and precise genetic repair in cells.

• Demonstrated ability to deliver genetic material to specific cells in non-human primates, with best-in-class preclinical data.

• Hypoimmune technology is used to hide transplanted cells from immune rejection, with applications in both diabetes and CAR T cell therapies.

Progress in type 1 diabetes therapy

• Developed gene-modified pluripotent stem cells (SC451) to create islets for curative therapy in type 1 diabetes, aiming for a single treatment with lifelong euglycemia and no immunosuppression.

• Achieved a milestone with a patient producing insulin after 40 years, published in the New England Journal of Medicine.

• Clinical strategy includes measuring C-peptide, insulin independence, and imaging to assess cell viability and durability.

• IND filing for SC451 planned for next year, with ongoing GLP toxicology and GMP manufacturing as key gating factors.

Clinical development and regulatory engagement

• Initial trials will target adults, expanding to adolescents and broader populations over time, with stepwise inclusion based on safety and efficacy.

• Regulatory interactions, especially with the FDA, have been constructive and recognize the therapy's transformative potential.

• Trial design will closely mirror successful Swedish study, focusing on replicating results with stem cell-derived product.