Sarepta’s gene therapy for Duchenne muscular dystrophy has produced stellar results in the first three patients treated in a clinical trial, raising hopes that it could halt or even reverse the muscle wasting disease.
The data were reported during Sarepta’s R&D update yesterday and – by the end of the day – shares in the US biotech had risen by a third, having been up almost 60% at one point. Analysts at Leerink said the results were a ‘home run’ and way ahead of expectations, and positions Sarepta as a “leader in this field.”
DMD – a rare muscle-wasting disease that mostly affects boys and typically is fatal by age 30 – is caused by a defect in the gene coding for dystrophin, a protein found in muscles that is critical for movement.
The data from the three boys – who appeared at the R&D event to rousing applause – showed that Sarepta’s AAVrh74.MHCK7 gene therapy led to the production of a truncated form of dystrophin at a level 38% of normal – which may not seem that impressive at first glance but according to clinicians could have a dramatic impact on the disease.
Read more: http://www.pmlive.com/pharma_news/sarepta_soars_on_positive_duchenne_data_1240923