Sarepta gene therapy study shows promise
Sarepta shares soar as gene therapy study shows promise
(Reuters) – Sarepta Therapeutics Inc shares jumped 60 percent on Tuesday after the drugmaker reported promising results from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD).
Data from the three-patient study showed that levels of serum creatine kinase (CK) — an enzyme strongly associated with muscle damage caused by DMD — fell 87 percent on average.
“I have been waiting my entire 49-year career to find a therapy that dramatically reduces CK levels and creates significant levels of dystrophin,” said Jerry Mendell, the study’s principal investigator said, referring to the protein that helps keep muscles intact.
DMD is a rare and progressive genetic disorder that hampers muscle movement, affecting one in every 3,500 to 5,000 males.
The disease occurs when an exon, or exons – a vital part of a gene – are absent at birth or get erased later, interfering with the composition of the remainder of the gene.
Sarepta already has approval for another DMD treatment, Exondys 51, and has been testing other therapies to treat the disease.
Sarepta’s shares rose as much as 67.7 percent to a record high of $176.60 on Tuesday.
Reporting by Manas Mishra in Bengaluru; Editing by Sai Sachin Ravikumar