Sana Biotechnology (SANA) investor relations material

Strategic focus and company identity

• Emphasis on engineering cells both ex vivo (for cell replacement) and in vivo (for cell repair or signaling change), maintaining a dual approach despite challenges in novel biology fields.

• Current focus is on Type 1 diabetes and in vivo CAR-T, with each representing distinct, uncorrelated risks and opportunities.

• Type 1 diabetes is highlighted as a large, underserved market with significant unmet need and life expectancy impact.

• In vivo CAR-T faces a dynamic competitive landscape, requiring speed and breadth in clinical development.

Type 1 diabetes program (SC451)

• Proof of concept achieved: gene-modified islet cells transplanted into a patient with no immunosuppression, producing insulin for 14 months.

• Manufacturing is progressing through three phases: phase I readiness, commercial launch scale, and large-scale production, with current focus on scientific challenges.

• IND filing for SC451 is targeted for this year, with both tech transfer and non-clinical testing nearing completion.

• Early clinical data will focus on safety, immune rejection, C-peptide levels, and insulin independence, with key milestones expected within a year.

• Partnership with Mayo Clinic aims to standardize clinical handling and ensure safe, replicable delivery of live cell therapies.

Competitive landscape and know-how

• Large market size in Type 1 diabetes means multiple players can succeed; learning from competitors and sharing field advancements is emphasized.

• Significant know-how required for gene editing and stem cell stability, with transparency about challenges and solutions.