Sana Biotechnology (SANA) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Key scientific advances and clinical data

• Demonstrated the first successful transplantation of gene-edited allogeneic islet cells in a type 1 diabetes patient without immunosuppression, resulting in endogenous insulin production after 30 years of insulin dependence.

• HIP platform enables transplanted islet cells to evade allogeneic and autoimmune rejection, confirmed by stable C-peptide levels and MRI imaging showing survival and function post-transplant.

• Achieved immune evasion by knocking out MHC class I and II and overexpressing CD47, preventing adaptive and innate immune rejection in humans.

• Phase 1 safety study met all endpoints, with no safety events and functional beta cells; patient will be followed for 15 years for long-term data.

• Preclinical data show HIP-modified islet cells persist and control blood glucose in mice for over 15 months.

Platform and pipeline expansion

• The hypoimmune (HIP) platform is being applied to B-cell-mediated autoimmune diseases (e.g., lupus, MS) with SC291, aiming for durable remission without ongoing therapy; GLEAM Phase 1 is ongoing.

• SC262, a HIP-modified CD22 CAR T, is in human testing for blood cancers in patients who failed CD19 CAR T therapy, with ongoing phase 1 VIVID trial.

• In vivo CAR T-cell technology using Fusogen enables cell-specific delivery and deep B-cell depletion in non-human primates and preclinical models without lymphodepleting chemotherapy.

• Manufacturing advances allow for scalable production, with hundreds of patient batches per run for allogeneic CAR T-cells.

• The company retains worldwide rights to all drugs and plans to partner for commercialization.

Manufacturing and operational challenges

• Major hurdle has been creating a GMP-compliant, genetically stable master cell bank; progress has been made, but clinical entry for SC451 will not occur within the next six months.

• Manufacturing scale-up is required to meet demand, with a target of producing trillions of cells for broad patient access.

• Conversion from research to GMP master cell bank involves rigorous documentation, sterility, and regulatory compliance.

• The company is working in parallel on new GMP banks to ensure readiness for clinical trials.

• Manufacturing and gene-editing challenges are being addressed to enable clinical and commercial scale.