Sarpeta Therapeutics has announced that the Food and Drug Administration (FDA – the drug regulator in the USA) has lifted the Clinical Hold that was issued to their phase I/II trial of gene therapy. The trial, which is taking place in the USA can now continue.
The trial aims to test the safety and effectiveness of delivering a small, but functional, dystrophin gene (micro-dystrophin) to the muscles using a harmless virus. Encouraging initial results were announced in June, but the FDA paused the trial in July after a routine test of the trial drug found pieces of DNA that should not have been present.
Sarepta quickly identified the cause of the issue and worked with the Research Institute at the Nationwide Children’s Hospital (where the trial is taking place) to come up with a plan to address it. The FDA has now approved the plan and the trial will continue as soon as possible.
The company still plans to start a larger trial of the gene therapy technology late this year and we’ll keep you updated as we hear more.
Find out more
- Contact Neil Bennett, Director of Research, to find out more on 020 7250 8240
- Book tickets for the Action Duchenne Conference
- Read Sarepta’s letter to the community
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