- Sarepta Therapeutics was up more than 57% Tuesday morning after a key trial showed its experimental gene therapy worked in three patients.
- Competitor Solid Biosciences gained 37%.
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Shares of Sarepta Therapeutics soaring, are up more than 57% Tuesday morning, after a key trial showed its experimental gene therapy worked in three patients.
Sarepta on Tuesday announced positive results for an early trial of three patients with duchenne muscular dystrophy who were given a gene therapy. After receiving the therapy, three boys, under the age of six, saw an increase in the protein. Duchenne muscular dystrophy is an inherited disorder in which a key protein is missing, leading to muscle weakness.
Competitor Solid Biosciences was up more than 37%, piggybacking the positive results.
"I have been waiting my entire 49-year career to find a therapy that dramatically reduces CK levels and creates significant levels of dystrophin," Dr. Jerry Mandell, who presented the positive results at the Sarepta's R&D Day, said in a press release. "Although the data are early and preliminary, these results, if they persist and are confirmed in additional patients, will represent an unprecedented advancement in the treatment of DMD."
Sarepta Therapeutics is up more than 205% this year.