Sonothera touts ultrasound gene-delivery platform for Duchenne, but experts remain skeptical
Sonothera, a California startup, says its ultrasound‑based technology can deliver therapeutic genes into cells, a method it believes could treat Duchenne muscular dystrophy and other genetic disorders. The company has shown striking results in animal studies, though it has not yet generated human clinical data.
The approach relies on focused ultrasound to temporarily open cell membranes, allowing gene‑editing vectors to enter. If successful, the technique could simplify delivery compared with viral vectors that dominate current gene‑therapy strategies.
However, leading researchers expressed doubt. Eric Olson of UT‑Southwestern called the claims "hard to believe," and Jeffrey Chamberlain of the University of Washington said they seem "too good to be true." Without human trial results, the scientific community remains cautious about the platform's real‑world potential.
Investors and observers will be watching for any forthcoming clinical data, which will be needed to move the concept beyond preclinical promise.
This writeup was produced by pharmadog from original reporting by STAT.
Original headline: “STAT+: Can ultrasound unlock the power of gene therapy? A startup makes big claims”
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