Access to treatments

Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy

Duchenne UK is delighted to share the news from Solid Biosciences that the company has announced the initiation of their first gene therapy clinical trial.

Enrollment is now underway at the first clinical trial site in the United States. Solid Biosciences is currently investigating potential sites in Europe.
The company has written an open letter to the Duchenne community, which contains details of the study. They have also provided a clear explanation of the science behind gene transfer. More information will be available shortly on Clinicaltrials.gov.
The company’s Investigational New Drug (IND)* application was cleared by the FDA, and the study has begun enrolling at the first site in the US.
In 2014, Duchenne UK partnered with the Duchenne Research Fund and Joining Jack to commit $5 million to help fund preclinical work for Solid’s gene therapy programme.
We are delighted that – just three years later – we are able to share this news with the community, and to thank the team at Solid Biosciences for their hard work and dedication to getting this programme into the clinic.

To read more on gene transfer and other clinical trials in this area please visit our website: Gene Therapy Overview
*An IND application is a request that companies submit to the Food and Drug Administration (FDA) in the US, to administer an investigational drug or biological product to humans.

Further further information please read: Solid Biosciences press release.

Published on 30 November 2017

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