Solid Biosciences advances gene therapy trials across muscle diseases

Solid Biosciences Inc. (NASDAQ: SLDB) reported progress across its gene therapy pipeline, with 33 participants dosed in its Duchenne muscular dystrophy trial and new clinical trials underway for Friedreich's ataxia and a cardiac condition.

The company's SGT-003 treatment for Duchenne muscular dystrophy has been dosed in 33 participants in the Phase 1/2 INSPIRE DUCHENNE trial as of January 9, 2026. The treatment has been generally well tolerated using a steroid-only prophylactic regimen, with outpatient dosing enabled since September 2025. No drug-induced liver injury, thrombotic microangiopathy or myocarditis has been observed as of the January cutoff date.

Solid enrolled the first participant in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled trial outside the U.S., with dosing expected in the first quarter of 2026. The trial has two active sites in Canada and Australia, with planned expansion to Europe beginning mid-2026.

The company dosed its first participant in the Phase 1b FALCON trial evaluating SGT-212 for Friedreich's ataxia. SGT-212 received FDA Orphan Drug designation and utilizes a dual route of administration approach. Initial data from this trial is anticipated in the second half of 2026.

For catecholaminergic polymorphic ventricular tachycardia, Solid has activated clinical trial sites for its ARTEMIS Phase 1b trial evaluating SGT-501, with participant screening underway. Initial safety data is expected in the second half of 2026.

The company has executed more than 50 agreements for use of its proprietary capsid technology AAV-SLB101, including licenses with corporations, institutions and academic laboratories. This capsid is designed to enhance targeting of skeletal muscle and cardiac tissue while reducing liver distribution.

Solid plans multiple interactions with the FDA during the first half of 2026 to align on Phase 3 trial design and confirmatory evidence requirements for potential accelerated approval of SGT-003.

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