Drug Profile

SGT 003

Alternative Names: SGT-003

Latest Information Update: 29 May 2025

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At a glance

Originator Solid Biosciences
Class Gene therapies
Mechanism of Action Dystrophin replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Duchenne muscular dystrophy
New Molecular Entity No

Highest Development Phases

Phase I/II Duchenne muscular dystrophy

Most Recent Events

15 May 2025 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children) in Italy (IV)
15 May 2025 Solid Biosciences plans to request a meeting with the US FDA to discuss potential accelerated approval pathways for SGT 003 in fourth quarter of 2025
06 Mar 2025 Updated pharmacodynamics data from a phase I/II INSPIRE DUCHENNE trial in Duchenne muscular dystrophy released by Solid Biosciences

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