Sana Biotechnology (SANA) investor relations material

Platform overview and therapeutic focus

• Focuses on immune-evasive cell therapies and in vivo delivery of genetic payloads, with lead asset targeting a functional cure for type 1 diabetes.

• Type 1 diabetes affects about nine million people globally, with significant unmet needs despite insulin therapy.

• Advances in islet transplantation and stem cell-derived islets have shown promise but are limited by supply and need for immunosuppression.

• Gene-modified islets transplanted without immunosuppression have shown persistent function in a human subject.

• IND submission for the lead program is targeted for next year, with clinical studies expected to begin in 2026.

Clinical data and development pathway

• Initial human data from a Swedish clinical study showed gene-modified islets survived and functioned without immunosuppression in one patient, with stable C-peptide and graft survival on imaging.

• The future product will use a master pluripotent stem cell line, gene-edited for immune evasion, enabling scalable manufacturing for millions of doses.

• Target dose for efficacy is around one billion cells per patient, based on field data.

• Regulatory alignment achieved; focus is on completing GMP manufacturing and GLP toxicology studies before human trials.

• Phase I trial is expected to enroll 12-15 patients, with manufacturing readiness and staggered dosing planned.

Manufacturing and technical challenges

• Major challenges include ensuring genomic stability and product purity during large-scale cell manufacturing.

• Genomic testing includes checks for on-target gene edits, chromosomal rearrangements, and cancer gene mutations.

• Years of work have gone into developing a stable, gene-edited cell line and aligning with global regulators on master cell bank release criteria.

• Cell viability post-transplant is expected to last decades, as seen in prior islet transplant fields.