Sangamo Therapeutics (SGMO) Corporate Presentation summary

Strategic focus and technology platforms

• Focused on developing genomic medicines for neurological diseases using zinc finger epigenetic regulation and advanced AAV capsid delivery platforms, enabling both intrathecal and intravenous brain delivery.

• Proprietary STAC-BBB capsid demonstrates industry-leading CNS tropism and robust blood-brain barrier penetration, validated in nonhuman primates and subject to multiple pharma partnerships.

• Zinc finger repressors (ZFRs) offer precise, potent, and specific gene regulation, with customizable potency and minimal immunogenicity due to human-derived components.

• SIFTER platform enables selection of neurotropic AAV capsids, supporting innovation in preclinical programs.

• Next-generation modular integrase (MINT) platform allows for large-scale, targeted genome editing without DNA breaks.

Pipeline and clinical progress

• Neurology pipeline includes wholly owned programs for idiopathic small fiber neuropathy (ST-503) and prion disease (ST-506), with ST-503 dosing expected in fall 2025 and preliminary efficacy data in Q4 2026.

• Prion disease program leverages STAC-BBB for intravenous delivery, with CTA submission anticipated mid-2026 and first-in-human trial planned for late 2026.

• Partnered programs target tauopathies, ALS/FTD, Huntington's disease, and other CNS indications, with agreements in place with Genentech, Astellas, Lilly, and others.

• Fabry disease program (isaralgagene civaparvovec) achieved positive topline results in the registrational STAAR study, with a BLA submission expected as early as Q1 2026.

• Hemophilia A program (giroctocogene fitelparvovec) met endpoints in Phase 3 AFFINE trial; seeking new commercialization partner.

Financial and business development highlights

• Raised $21 million in net proceeds from an equity offering in Q2 2025; cash and equivalents of $38.3 million as of June 30, 2025, expected to fund operations into Q4 2025.

• $88 million received from partners to date, with up to $6.1 billion in potential future milestones and exercise fees.

• Actively engaged in business development discussions for pipeline and platforms, with ongoing negotiations for Fabry commercialization and potential new STAC-BBB partnerships.

• Non-GAAP operating expenses for Q2 2025 were $33 million; 2025 guidance is $125–$145 million, assuming adequate funding.