In recent press releases, Sarepta Therapeutics has given updates on their ongoing clinical trial programmes. These include exon skipping and gene therapy trials for Duchenne.
Exon Skipping
Sarepta has announced that the first participant in the Phase 2 trial of SRP-5051 has received the trial drug. Designed to skip exon 51 of the dystrophin gene, SRP-5051 is a second generation exon skipping drug . Researchers believe it will enter muscles more efficiently than the first generation drugs and this could improve its efficiency.
The trial is in 2 parts. The first will identify the maximum dose of SRP-5051 that participants can tolerate. In the second part, all participants will receive that dose of SRP-5051 for 24 weeks.
Gene therapy
Sarepta has announced that a participant in the company’s Phase 2 gene therapy trial experienced rhabdomyolysis. This is a condition where large numbers of muscle fibres breaks down quickly. Without treatment, it can potentially cause kidney problems.
Everybody living with with Duchenne has an increased risk of rhabdomyolysis. The event therefore may or may not be related to the trial. The trial is “blinded”, so clinicians and participants don’t know whether the child received the potential treatment or a placebo. It’s therefore impossible to say at this point whether the event is related to the gene therapy. Importantly, the event was reviewed by the trial’s independent safety board, which gave permission for the trial to continue.
Sarepta are already planning a further gene therapy trial, which they hope to start early next year.
Find out more
- Contact Neil on 02072508240
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