Sana Biotechnology (SANA) investor relations material

Strategic focus and scientific progress

• Prioritized overcoming allogeneic rejection in cell therapy and efficient genetic payload delivery, with a focus on type 1 diabetes as a major unmet need.

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

• Human data show transplanted, gene-edited islets can survive and function for over a year without immunosuppression, with no adverse immune response detected.

• Manufacturing process advanced to GMP master cell bank, with ongoing tech transfer to CDMO and alignment with global regulators.

• IND filing and phase I trial initiation targeted for this year, with rapid proof-of-concept expected due to early measurable outcomes.

Clinical development and regulatory engagement

• Phase I trial will enroll a broad adult type 1 diabetes population, expanding to younger and higher-risk groups over time.

• Study will be conducted in the U.S. and other geographies, with straightforward trial design and focus on safety and immune evasion.

• Ongoing non-clinical work includes efficacy and GLP-tox studies, with close and frequent engagement with the FDA and other regulators.

• Regulatory agencies recognize the unmet need and transformative potential, providing clear guidance and access for program advancement.

• Manufacturing for phase I is in tech transfer, with scalability and genomic stability as key challenges for commercial readiness.

In vivo CAR T platform and future plans

• Progress made in developing in vivo CAR T cells, aiming to eliminate conditioning chemotherapy and democratize access.

• Platform uses a novel, highly specific fusogen for targeted gene delivery, minimizing off-target effects and immunogenicity.

• Preclinical studies in non-human primates show dose-dependent CAR T expansion, B cell depletion, and safety, with no off-target effects.

• First human trials for in vivo CAR T expected this year, with data anticipated in 2026; initial focus on cancer, then rapid expansion to autoimmune diseases.

• Partnership sought for broader development of CAR T assets, with preference to secure initial human data before out-licensing.