Sana Biotechnology (SANA) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic focus and scientific progress

• Two main goals: overcoming allogeneic rejection in cell therapy and delivering genetic payloads to cells, with a current focus on type 1 diabetes and CAR T cell therapies.

• Demonstrated ability to transplant gene-edited cells that evade immune rejection, with a master cell bank established and regulatory alignment achieved.

• In vivo CAR T platform aims to democratize access by eliminating conditioning chemotherapy and enabling off-the-shelf therapies, with first human data expected in 2026.

• Type 1 diabetes remains a large unmet need, with the goal of a one-time, insulin-independent, immunosuppression-free cure.

• Published clinical data show gene-edited islets can survive and function in humans without immunosuppression, supporting the potential for a scalable cure.

Clinical and regulatory milestones

• First-in-human study demonstrated safety and immune evasion of gene-edited islets, with ongoing C-peptide production and no adverse immune response after one year.

• Master cell bank created from a universal donor stem cell, enabling scalable production of islets for therapy.

• IND filing and phase I trial initiation targeted for this year, with rapid proof-of-concept expected within months of patient dosing.

• Regulatory agencies, including the FDA, are highly engaged due to the unmet need and transformative potential, with frequent and constructive dialogue.

• Phase I trial will enroll a broad adult population in the US and internationally, with plans to expand to younger and higher-risk patients over time.

Manufacturing and commercialization challenges

• Manufacturing for phase I is underway, with tech transfer in progress and ongoing work to ensure purity and genomic stability at scale.

• Scaling to commercial production for a disease affecting millions presents significant scientific and capital challenges, particularly in increasing doses per manufacturing run.

• Investment in manufacturing will be ongoing, with much of the scale-up occurring post-approval.

• Organizational resilience is emphasized to navigate inevitable development hurdles.