Muscular Dystrophy

Pipeline

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Pliant is developing therapies that may directly improve muscle function and viability while reducing the reactive fibrosis that results from progressive muscle destruction.

Our Muscular Dystrophy Program

Muscular dystrophy comprises a group of inherited diseases, all characterized by inborn errors in dystrophin, a protein that is crucial to muscle function. Lack of proper dystrophin function results in damage to muscle cells, which over time leads to muscle degeneration, and replacement of muscle tissue with fat and fibrotic tissue.

Pliant has identified a target integrin receptor that acts as a natural compensatory mechanism in Duchenne muscular dystrophy (DMD), as well as other types of muscular dystrophy. By activating this receptor, it may be possible to stabilize the muscle cells, thereby increasing muscle strength and reducing muscle damage.

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Program Highlights

  • Targeting an integrin receptor on the muscle cell surface
  • Integrin target is upregulated as a compensatory mechanism in multiple types of muscular dystrophy
  • Acts as a substitute for dystrophin, helping to stabilize the muscle membrane, increasing muscle strength, and decreasing muscle damage
  • Mutations in the target receptor alone actually causes a human congenital myopathy similar to muscular dystrophy
  • Potential to combine across multiple muscular dystrophy indications because the target integrin is upregulated across multiple forms of muscular dystrophy

Development Status

  • This program is currently in preclinical development

Our Advanced Integrin Library

At Pliant, we have developed an industry-leading library of over 7,000 annotated integrin-binding molecules to probe the molecular drivers of fibrotic disease. With this library, we screen for activity against newly identified targets, to create a deep well of potential drug candidates.

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