Sonothera's Gene Therapy Claims Draw Expert Skepticism
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- Sonothera, a small California startup, has spent the last year claiming its technology could potentially treat Duchenne muscular dystrophy and possibly a slew of other genetic diseases.
- Sonothera does not yet have any clinical data, instead leaning on animal study results described as so stunning that outside experts struggle to accept them.
- Eric Olson, molecular biology chair at UT-Southwestern Medical Center, said of Sonothera's findings: "I find it hard to believe."
- Jeffrey Chamberlain, a longtime Duchenne gene therapy expert at the University of Washington, said the company's claims "seem a bit too good to be true."
- Two of the field's most prominent Duchenne researchers have publicly cast doubt on preclinical data that the small startup is using to position itself as a potential cure for multiple genetic diseases.
Why it matters: Two of the field's most prominent Duchenne gene therapy researchers — Eric Olson of UT-Southwestern and Jeffrey Chamberlain of the University of Washington — have publicly questioned Sonothera's preclinical findings, raising the stakes for a company that hasn't yet entered clinical trials but is already making bold claims about curing Duchenne and other genetic diseases.
