Update on Gene TherapyJuly 3, 2018 12:53 pm
In recent years, our community has watched with hope as pre-clinical data has emerged showing the promise of gene therapy as a valuable treatment for everyone living with Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy is a genetic condition and is caused by a lack of protein called Dystrophin that is not produced by those that have Duchenne.
The year began with much hope with gene therapy: THREE companies, Solid Biosciences, Sarepta and Pfizer, began dosing patients in separate clinical trials using this approach. And this week has seen exciting developments.
Solid Biosciences announced that the U.S Food and Drug Administration (FDA), had lifted the clinical hold on IGNITE DMD, the company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer for the treatment of DMD. Solid Biosciences has dosed one patient so far and they are now free to continue to dose patients. – Alex’s Wish invested in this alongside Duchenne Research Fund and Duchenne UK who invested over $5million
Sarepta Therapeutics announced positive preliminary results from 3 of its patients dosed in its Phase 1/2a gene therapy clinical trial, conducted by Jerry Mendell M.D of Nationwide Children’s Hospital. All three children showed that they were producing micro-dystrophin in their muscle fibers. They also saw a significant drop in an enzyme known as creatine kinase (CK) which is indicative of muscle damage. No serious side effects were seen.
These are encouraging strides forward for us all. There is still a lot of work to do to ensure the success of all these treatments and their eventual availability to as many children and young adults with Duchenne as possible. We will continue to update you when there is further news.