Prosensa announces exon skipping progress

Muscular Dystrophy Campaign 

 Tuesday 23 October 2012  

Prosensa announces exon skipping progress

Dutch drug company Prosensa has announced progress on the development of new molecular patches which may have the potential to treat boys with Duchenne muscular dystrophy. The company hopes two more molecular patches will enter clinical trial in the next six months and has begun pre-clinical testing of a further two molecular patches. With two molecular patches already in clinical trials, in total, the six molecular patches could have the potential to treat 40% of boys with Duchenne muscular dystrophy.

Prosensa is a Dutch drug company working with GlaxoSmithKline to develop exon-skipping technology to treat boys with Duchenne muscular dystrophy. The companies are currently testing molecular for exons 44 and 51 of dystrophic in clinical trials.

In a press release, Prosensa has announced  that molecular patches for exons 45 and 53 have been granted "orphan drug" status in the European Union. "Orphan drug" status is given to candidate drugs with the potential to treat rare diseases. The company hopes the status will allow them to develop these potential drugs more quickly and aims to start clinical trials in the next six months. Prosensa also announced that they have started preclinical work on molecular patches for exons 52 and 55 of dystrophin.

In total, these six molecular patches have the potential to treat 40% of boys with Duchenne muscular dystrophy. However, like all exon skipping technology, the potential treatments will not cure Duchenne muscular dystrophy, but will reduce the severity of the symptoms to those seen in people with Becker muscular dystrophy.