Sana Biotechnology (SANA) Morgan Stanley 24th Annual Global Healthcare Conference summary

Strategic vision and recent progress

• Focused on engineering cells to address highly prevalent diseases using scalable platforms, with two main programs advancing toward clinical studies soon.

• Lead program aims for a one-time functional cure for type 1 diabetes using gene-modified, iPSC-derived islets, with IND clearance and phase I study targeted for this year.

• Collaboration with Mayo Clinic has improved product delivery, storage, and reproducibility, and included a $25 million investment.

• Regulatory interactions have been transparent and supportive, with global interest in translating early human data to broader populations.

• Capital allocation is primarily directed to the diabetes program, with $160 million in cash runway into mid-next year and plans to seek additional funding as needed.

Type 1 diabetes program details

• Demonstrated gene-modified islets can survive and function in humans without immunosuppression for at least 14 months.

• Phase I/II trial will enroll around a dozen patients, focusing on safety, immune evasion, efficacy, and consistency of results.

• Key safety concerns include short-term hypoglycemia and long-term tumor risk, with a safety switch engineered into the cells.

• Manufacturing challenges addressed by creating a genomically stable GMP master cell bank and transferring processes to GMP suites.

• Broad adult patient population targeted, with plans to expand to pediatric patients if successful.

In vivo CAR T platform (SG293)

• SG293 leverages a Fusogen platform for in vivo CAR T generation, initially targeting non-Hodgkin lymphoma, with potential expansion to autoimmune diseases.

• Platform emphasizes specificity to T-cells, chromosomal integration of CAR DNA, and separation of genetic entry from T-cell activation to reduce toxicity.

• Investigator-initiated trial in China expected to yield data in the first half of 2027, delayed due to regulatory caution after unrelated adverse events.

• Early proof of concept will focus on safety and complete responses with undetectable ctDNA, aiming to match or exceed autologous CAR T performance.

• Second BCMA-targeted drug is ready but will advance only after initial platform validation due to capital constraints.