Pipeline & Programs:Pipeline

Phase I/II

Molecule Biologic

Mechanism In Duchenne, 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 is believed to retain its key components and functionality. In preclinical models, therapeutic administration of microdystrophin by adeno-associated virus (AAV) has been shown to stabilize the DGC and restore muscle function.

Impact on Duchenne 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 the type of dystrophin gene mutation.

Preclinical

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. We believe this may be accomplished by targeting a protein known as the latent TGF-β binding protein 4 (anti-LTBP4).

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