SGT-003

SGT-003

Status

Phase |

Therapeutic Approach

Restoring or Replacing Dystrophin

SGT-003 is an adeno-associated virus (AAV) mediated gene transfer that contains an engineered version of the dystrophin gene (microdystrophin). SGT-003 uses a novel AAV capsid designed by Solid Biosciences. The microdystrophin gene in SGT-003 has been designed to produce a functional form of dystrophin protein containing the nNos domain in skeletal and cardiac muscles.

Status

A Phase 1 trial is not yet recruiting.

Sponsor

This program is sponsored by Solid Biosciences.

Related Studies

NOT YET RECRUITING
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

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