Sangamo Therapeutics (SGMO) shares were up premarket Tuesday after the company reported topline results from a phase 1/2 study evaluating ST-920 for the treatment of Fabry disease and said the gene therapy candidate showed a favorable safety and tolerability profile.
Following a single dose, ST-920 demonstrated a "positive" mean annualized estimated glomerular filtration rate, or eGFR, slope at 52-weeks across all dosed patients, the company said.
Sangamo said that the Food and Drug Administration agreed that the eGFR slope would serve as an intermediate clinical endpoint under an accelerated approval pathway. GeFR is a key indicator of kidney function decline.
Looking ahead, the genomic medicine company said it intends to submit a biologics license application under an accelerated approval pathway as early as Q1 2026.
Shares of the company were 6% higher ahead of Tuesday's opening bell.
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