Sana Biotechnology (SANA) TD Cowen 46th Annual Health Care Conference summary

Strategic focus and milestones

• Targeting immune evasion for cell therapies and in vivo delivery of genetic payloads, with significant progress in both areas and key data expected within 12–24 months.

• Lead program aims for a one-time curative therapy for type 1 diabetes using gene-modified, stem cell-derived pancreatic islets, with regulatory filings and clinical studies planned in the near term.

• Three value inflection points: cell engraftment and immune evasion (weeks), restoration of normal glucose without insulin (2–6 months), and replicability across patients (within 24 months).

• In vivo CAR T-cell program for blood cancers to start clinical studies this year, with early efficacy and safety data anticipated within 12 months.

• Manufacturing scalability, regulatory alignment, and commercial model development are identified as critical future challenges.

Scientific and competitive landscape

• Approach to immune evasion involves knocking out MHC class I/II and overexpressing CD47, inspired by red blood cell biology, to avoid both adaptive and innate immune rejection.

• Competitors use alternative proteins (e.g., CD300a) and strategies, but the field remains open with room for multiple successful approaches.

• Manufacturing process starts from a genomically stable, gene-edited master cell bank, with robust quality controls and regulatory alignment.

• Intramuscular transplantation is chosen for safety, scalability, and monitoring advantages over portal vein delivery.

• Broad patient eligibility is planned, starting with adults and expanding to children, leveraging an O negative donor to avoid blood type matching.

Clinical development and disclosure plans

• IND-enabling activities include completion of GLP toxicology studies and tech transfer to GMP manufacturing, with plans to file within 2026.

• Disclosure of milestones will focus on material events, such as IND clearance and key clinical data, rather than every step.

• Early clinical endpoints include insulin independence and real-time glucose monitoring, with dose selection guided by regulatory alignment and competitive benchmarks.

• Scaling manufacturing for broader access will be addressed in stages, starting with phase I supply and progressing to commercial launch.

• In vivo CAR T-cell program leverages a virus-like particle platform for targeted, integrated delivery, aiming for improved specificity and safety over mRNA-based competitors.