Corrective Therapies

Our corrective therapies platform is dedicated to the development of gene transfer candidates for Duchenne muscular dystrophy.

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Innovating in Gene Transfer

Solid chose to focus initial efforts on gene transfer due to the significant progress in our understanding of the dystrophin gene, recently reported successes in gene therapy clinical development and advances in manufacturing.

Facts Important for Gene Transfer Development

Microdystrophin Gene Transfer

Solid’s lead program is an adeno-associated virus (AAV) microdystrophin gene transfer candidate, which aims to enable the systemic delivery of a synthetic, functional version of the dystrophin gene. We are also actively exploring other potential gene therapy candidates that could be beneficial for Duchenne muscular dystrophy (DMD).

"We believe our approach is largely unprecedented, yet grounded in nearly twenty years of research."

– Joel Schneider, Ph.D., Chief Technology Officer and Head of Exploratory R&D

The vehicles that enable widespread delivery of these genes to the body are small viruses, derived from adeno-associated viruses or AAV. These viruses are not known to cause diseases and are currently being explored in many disorders for their potential to deliver therapeutic genes. However, these viruses do not have the capacity to carry the entire dystrophin gene.

Over the past two decades, efforts to minimize the size of dystrophin have resulted in microdystrophin genes that are believed to retain the most critical components of the larger version, yet are small enough for the AAV to carry.

  • Image 1: In DMD, the dystrophin protein is absent due to a mutation in the dystrophin gene, causing muscle cells to deteriorate.
    Image 1: In DMD, the dystrophin protein is absent due to a mutation in the dystrophin gene, causing muscle cells to deteriorate.
  • Image 2: In healthy individuals, the dystrophin gene makes a functional dystrophin protein, which is essential for  muscle function and structure.
    Image 2: In healthy individuals, the dystrophin gene makes a functional dystrophin protein, which is essential for muscle function and structure.
  • Image 3: AAV-mediated gene transfer may be a meaningful way to replace the mutated dystrophin gene. However, the natural dystrophin gene is too big to fit into AAV.
    Image 3: AAV-mediated gene transfer may be a meaningful way to replace the mutated dystrophin gene. However, the natural dystrophin gene is too big to fit into AAV.
  • Image 4: Microdystrophin gene is a smaller  version of the dystrophin gene that retains essential functional elements yet is small enough to fit into AAV.
    Image 4: Microdystrophin gene is a smaller version of the dystrophin gene that retains essential functional elements yet is small enough to fit into AAV.
  • Image 5: Carrying the microdystrophin gene, the AAV can then be administered systemically to the body.
    Image 5: Carrying the microdystrophin gene, the AAV can then be administered systemically to the body.
  • Image 6: If successful, the body will utilize the newly produced microdystrophin protein similarly to dystrophin, resulting in a healthier muscle function.
    Image 6: If successful, the body will utilize the newly produced microdystrophin protein similarly to dystrophin, resulting in a healthier muscle function.

Solid chose to pursue development of SGT-001 because of its potential to drive the expression of a modified but functional dystrophin protein, regardless of specific genetic mutation. Results from two preclinical studies have demonstrated that a single administration of SGT-001 led to long-term expression of the microdystrophin protein in muscle, as well as improvements in muscle histology and function. 

The safety and efficacy of SGT-001 are currently being evaluated in a Phase I/II clinical trial. 

Why Gene Transfer?

Gene transfer is an investigational approach that aims to significantly slow or even halt the progression of DMD by delivering a modified yet functional dystrophin gene throughout the body.

Scientists have been exploring the potential of using genes as medicine to treat disease for more than 35 years. Several recent advancements have made it possible to apply this technology to DMD.

A Different Way to Restore What’s Missing

Gene transfer is potentially beneficial in DMD because it aims to address the root cause of the disease. While the risks and benefits of gene transfer in DMD still need to be evaluated in human clinical trials, early research is encouraging.

  • Potential to Help Many

    Because gene transfer targets the underlying cause of DMD, it has the potential to benefit those living with the disorder, regardless of a person’s disease mutation.

  • Potential to Slow or Stop DMD

    Gene transfer may be a way to restore expression of a modified yet functional dystrophin in all important muscle groups, including the heart, diaphragm and skeletal muscles.

  • Potential Impact

    While gene transfer is intended to promote ongoing expression of a modified dystrophin after a single treatment, the duration of its potential effect is unknown and needs to be evaluated in clinical studies. 

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