With Its First Human Data, Solid Slips in Duchenne Gene Therapy Race

The race to treat Duchenne muscular dystrophy with gene therapy, a one-time, potentially long-lasting treatment, is being closely watched. But new data from Solid Biosciences cast doubt on whether the Cambridge, MA, company can keep pace with the current leader, Sarepta Therapeutics.

Solid (NASDAQ: SLDB) this morning is disclosing the first results from the first three Duchenne patients in a study known as IGNITE DMD, an early-stage trial testing its experimental gene therapy, SGT-001. To be clear, the results are early—from biopsies taken three months after treatment—and at just the lowest dose of the gene therapy Solid plans to… Read more »

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