Solid Biosciences announce FDA have removed clinical hold from their gene therapy trial

Solid Biosciences have announced that the FDA has given approval for the company to restart the phase I/II IGNITE DMD clinical trial. The clinical trial, which aims to test the safety and effectiveness of a potential treatment called SGT-001, was placed on hold after the first person to receive the potential drug experienced unexpected side effects.

SGT-001 uses a harmless virus to deliver a functional copy of the dystrophin gene to cells. However, since the gene is so large – too big to be carried by the virus – researchers have developed smaller genes that can produce smaller but functional versions of the dystrophin protein (called microdystrophin). By using a virus that preferentially infects muscle cells, researchers hope to be able to restore some dystrophin production where it is needed most.

The clinical trial of the micro-dystrophin carrying virus will now continue to recruit participants (approximately 16) in Florida and will use a slightly changed . To learn more about the trial design, you can read more in Solid Biosciences’ letter to the community here.