Sana Biotechnology (SANA) 2024 Wells Fargo Healthcare Conference summary

Platform and technology overview

• Focused on cell and gene therapy with curative intent, addressing capital intensity and accessibility challenges.

• Developed a platform for in vivo delivery of RNA, DNA, or protein payloads to specific cells, enabling new categories of medicine.

• Advanced cell-specific delivery and hypoimmune (cloaking) technology to enable engraftment, function, and persistence of therapeutic cells.

• Overcomes immune rejection by knocking out MHC Class I/II and overexpressing CD47, validated in animal models and early human data.

• Four clinical programs in seven indications, with early human data showing promising responses in lymphoma and leukemia.

Clinical program highlights

• Three main clinical areas: Type 1 diabetes, B-cell-mediated immune diseases (e.g., lupus, MS), and blood cancers (lymphoma, leukemia).

• Type 1 diabetes program aims for insulin independence without immunosuppression from a single cell therapy treatment.

• B-cell disease program leverages allogeneic CAR T-cells for scalable, predictable therapy; first clinical data expected this year.

• Oncology program includes CD19 and CD22 allogeneic CAR T-cells, with early data showing complete and partial responses.

• Phase 1 studies underway in autoimmune and oncology indications, with dose escalation and patient enrollment ongoing.

Type 1 diabetes program details

• Investigator-sponsored trial uses gene-modified cadaveric islet cells to test immune evasion without immunosuppression.

• Key readouts include cell survival (via MRI), C-peptide production, and improved blood glucose control.

• Results from a single patient could be definitive; if successful, would generalize to broader applications.

• Manufacturing challenges include ensuring adequate CD47 expression and genomic stability in stem cell-derived islets.

• Internal SC451 program progressing, with focus on master cell bank stability, cell purity, genetic modification, and scalable production.