Key results of the $SGMO STAAR study
Positive eGFR slope at 52 weeks: Mean +1.965 mL/min/1.73 m²/year (95% CI: –0.153 to 4.083) in all 32 treated patients – confirmed as the primary endpoint by the FDA for accelerated approval
Long-term data over 104 weeks: continues to be positive (+1.747 mL/min/1.73 m²/year; CI: –0.106 to 3.601) in 19 patients
Secondary endpoints:
Stable α-gal-A activity for up to 4.5 years;
All 18 patients who previously received enzyme replacement therapy (ERT) are independent of it;
Lipid markers (Lyso-Gb3) largely stable;
Improvements in cardiac parameters, quality of life (SF-36), pain reduction, physical function, sweating
Safety profile: Predominantly Grade 1/2 side effects (fever, headache, nasopharyngitis); no treatment discontinuations due to safety.
@BiotechAnalysst In summary, this image shows details for an upcoming international oral presentation on June 20, 2025, titled "Health-Related Quality of Life and Joint Health with Giroctocogene Fitelparvovec Gene Therapy," to be held in Washington D.C., USA, at the International Society on...
Join our CEO, Sandy Macrae, for a fireside chat at the H.C. Wainright 6th Annual Neuro Perspectives Hybrid Conference on Tuesday, June 17th at 7:00 a.m. EDT.
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🔬 ICYMI: Exciting progress was highlighted last week during the prestigious #PresidentialSymposium at #ASGCT2025, showcasing promising data for the potential treatment of prion disease. To view this presentation and more Sangamo content from ASGCT, https://t.co/EkI1IrwToJ