Sana Biotechnology (SANA) investor relations material

Strategic vision and platform overview

• Focus on modulating genes and using cells as medicines, targeting immune rejection and in vivo delivery challenges.

• Progress in developing hypoimmune technology to hide transplanted cells from immune detection, with initial focus on Type 1 diabetes.

• In vivo delivery platform aims for cell-specific, scalable, and repeatable gene editing, with CAR T cell therapies as a lead application.

• Both platforms have shown promising preclinical and early clinical results, with plans to expand indications if successful.

• Forward-looking statements include anticipated clinical data and regulatory filings within the year.

Hypoimmune platform and Type 1 diabetes program

• Gene edits include knockout of MHC Class I and II and overexpression of CD47 to evade both adaptive and innate immune responses.

• Validated in vitro, in animal models, and in a human patient with Type 1 diabetes, showing cell survival and function for over a year.

• Patient data demonstrate production of C-peptide, insulin response to meals, and cell visibility via PET-MRI, with no immune rejection.

• Manufacturing advances include creation of a genomically stable master cell bank for scalable stem cell-derived islet production.

• IND filing and phase one clinical trial for the stem cell-derived islet product targeted for this year, with global regulatory alignment.

Safety, scalability, and regulatory considerations

• Safety measures include genomic stability, product purity, and early detection systems for adverse events.

• Multiple safety mechanisms: surgical removal, embedded kill switch, and antibody-mediated cell elimination.

• Long-term patient follow-up (minimum 15 years) planned per regulatory requirements.

• Scalability addressed by master cell bank and robust manufacturing processes to meet large patient populations.

• Regulatory discussions indicate readiness for clinical advancement and product release.