Developing therapies for Duchenne muscular dystrophy

Our Pipeline

Solid is building a portfolio with the potential to address the many facets of the disease.

Corrective Therapies

SGT-001
Microdystrophin Gene Transfer

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Molecule: Biologic

Mechanism: In DMD, the absence or near-absence of the protein dystrophin leads to muscle membrane instability and disruption of the dystrophin glycoprotein complex (DGC). Microdystrophin is a synthetic version of the dystrophin gene that retains its key components and functionality. In preclinical models, its therapeutic administration by adeno-associated virus (AAV) has been shown to stabilize the DGC and rescue muscle function.

Impact on DMD: The large size of the dystrophin gene has historically prevented direct replacement as a therapeutic strategy. Preclinical studies have shown that microdystrophin AAV-mediated gene transfer enables systemic delivery of the truncated gene and has the potential to slow or halt disease progression, regardless of dystrophin gene mutation.

Disease-Modifying Therapies

SB-001
LTBP4

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Molecule: Biologic

Mechanism: Muscular dystrophy is characterized by the replacement of muscle with non-functional fibrotic tissue, which is caused by inflammation and muscle degeneration. The goal of this program is to target a known mediator of fibrosis, TGF-β,  and inhibit its activity. This may be accomplished by targeting a protein known as the latent TGF-β binding protein 4 (LTBP4).

Impact on DMD: LTBP4 has been identified as a genetic modifier of DMD and has been associated with a delayed loss of ambulation. Through the ability of this protein to reduce TGF-β activity, LTBP4 activation has the potential to reduce fibrosis in DMD patients and slow functional decline.

Assistive Devices

Solid Suit

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Description: Soft wearable device that can be individualized to allow patients greater mobility and help preserve muscle function.

Impact on DMD: DMD leads to progressive and irreversible muscle loss.  Even with therapeutic intervention, many patients will still require significant physical assistance.  By creating a soft, breathable suit that can be tailored to individual needs, patients may be able to perform daily life activities with greater ease.

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