Sangamo Therapeutics (SGMO) Barclays 27th Annual Global Healthcare Conference 2025 summary

Company overview and strategic focus

• Genomic medicine company advancing two BLAs in 2024 for Fabry disease and Hemophilia A.

• First-in-class Nav1.7 gene therapy repressor entering clinic in summer for neuropathic pain.

• Proprietary capsid technology licensed to other companies, with plans for prion disease trial in 2026.

• Focused on neuroscience and neurology using zinc finger repressors and advanced delivery tech.

Fabry disease program updates

• Filing for accelerated approval planned for second half of 2024, with launch targeted for second half of 2025.

• Statistically significant improvements in eGFR, quality of life, pain, and renal function observed in clinical data.

• 18 patients discontinued ERT, with some off treatment for over three years and sustained enzyme levels.

• Deal announcement for Fabry asset may be delayed to Q2 2024 but is considered urgent for funding.

• FDA requires one-year data for 32 patients for accelerated approval; two-year data may support full approval.

Financial and partnership outlook

• Cash runway extends through Q1 2025, with additional capsid deals under discussion to extend runway.

• Multiple parties in late-stage discussions for Fabry asset partnership.

• Funding from Fabry deal is critical to advance other pipeline projects.