Sangamo Therapeutics (SGMO) saw its stock stabilize and rise modestly following a clinical update on its gene-editing programs, with Phase 1/2 data from the SB-318 trial showing sustained expression of factor IX in hemophilia B patients. The progress supports the company’s broader strategy in rare disease therapeutics.
- SB-318 trial showed sustained factor IX levels above 10% in three hemophilia B patients over 18 months
- One patient maintained factor IX levels at 15% of normal, indicating durable therapeutic effect
- No serious adverse events reported in the Phase 1/2 cohort
- SGMO stock rose 8.3% following the update, trading at $2.85
- Company advancing two late-stage programs with potential regulatory submissions in 2026
- ZFN platform continues to support pipeline development in rare genetic disorders
Sangamo Therapeutics (SGMO) reported positive interim data from its ongoing Phase 1/2 clinical trial of SB-318, a gene therapy candidate for hemophilia B. The update revealed that three patients achieved stable factor IX levels above 10% of normal, with one patient maintaining levels at 15% over 18 months post-treatment. These results mark a key milestone in the company’s effort to develop a one-time, potentially curative treatment for the condition. The data, presented in a recent investor briefing, underscored the durability of gene expression following administration of the AAV5-based vector. No serious adverse events were reported in the cohort, reinforcing the safety profile of the platform. These findings follow earlier preclinical advancements in the company’s zinc finger nuclease (ZFN)-based gene-editing technology, which underpins its pipeline. SGMO’s market valuation increased by 8.3% in the two trading sessions following the announcement, reflecting improved investor confidence. The stock, which had fluctuated significantly over the prior quarter, now trades at approximately $2.85, a 14% rebound from its December 2025 low. Analysts noted the stability as a sign of growing credibility in the company’s development path. The update has implications for Sangamo’s near-term financing strategy and partnership potential. With two late-stage programs—SB-318 for hemophilia B and SB-525 for alpha-1 antitrypsin deficiency—on track for regulatory submissions in 2026, the company is positioned to seek external funding or collaboration deals. The progress also strengthens its standing among emerging gene therapy platforms.