Sarepta Therapeutics has announced impressive preliminary results from their gene therapy trial.
Results from the first three patients, 90 days after treatment, have been released. Strong dystrophin production was seen in the boy’s muscles and large reductions in creatine kinase levels in the blood were seen. Creatine kinase is an enzyme that leaks into the blood when muscle damage occurs. The safety profile also appears to be good so far.
This gene therapy involves using a virus to deliver a shortened, but functional, version of the dystrophin gene into the body. Dystrophin is the gene that is faulty in those with Duchenne. The trial is being carried out at Nationwide Children’s Hospital in Ohio. Two other gene therapy trials are also underway in the US for Duchenne – conducted by Solid Biosciences and Pfizer.
- More information about Duchenne see https://www.saveoursons.org.au/what-is-dmd/
- Read the Sarepta press release http://investorrelations.sarepta.com/static-files/d58018c9-603a-4058-8f68-f328dd53f12f