Developing therapies for Duchenne muscular dystrophy

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360-degree approach to targeting Duchenne muscular dystrophy

Solid Biosciences is singularly focused on solving Duchenne muscular dystrophy and meeting the diverse needs of patients, from addressing the underlying cause of the disease to managing its multiple manifestations.

Our mandate is to accelerate the discovery and development of multiple scientific approaches in parallel as quickly and safely as possible. We have established collaborations with industry, academia and non-profits – all with the goal of moving closer to effective therapies.

We are building a diversified portfolio of targeted therapeutic candidates. All assets go through an extensive diligence process, culminating with a commitment to identify a path forward.

Scientific Pillars

Our 360-degree approach to discovering and developing therapies for DMD has led us to organize our programs across several scientific pillars:

  • Corrective Therapies

    Gene transfer has the potential to address the genetic cause of Duchenne muscular dystrophy, the absence or near-absence of a critical muscle structural protein called dystrophin.  If successful, gene transfer has the potential to significantly slow or even halt the progression of the disease.

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  • Disease-Modifying Therapies

    Targeting the multiple manifestations of Duchenne muscular dystrophy is important to limit the impact of dystrophin absence on muscle health and function.

    As a platform-agnostic company, Solid is examining disease-modifying candidates that are intended to help reduce the impact of Duchenne muscular dystrophy.

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  • Disease Understanding

    Measuring disease progression and the impact of therapeutic candidates in Duchenne muscular dystrophy is difficult because of the disease's complexity. Biomarkers may offer a solution to clarify underlying disease biology and more effectively evaluate therapeutic interventions.

    Accurate biomarkers have the potential to accelerate the discovery and development of meaningful therapeutic advances. 

  • Assistive Devices

    Solid Biosciences is proudly partnered with Silicon Valley's Superflex Inc., Parent Project Muscular Dystrophy, and other engineering and disease experts to develop assistive devices that may help patients perform everyday activities with greater ease.

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Start with the problem

Solid is different from other life science companies because we ask: “What approaches can address the problems that Duchenne muscular dystrophy patients face?,” rather than: “What problem can I address with my approach?”

Classical drug development begins with a scientific discovery that is translated into a therapeutic approach – such as the identification of a new method for increasing or decreasing the activity of a specific protein. This discovery is then tested in potentially suitable indications to better understand where the technology may offer benefit.

Our process is designed to explore numerous methodologies and approaches that may benefit Duchenne muscular dystrophy patients. This process allows us to think critically about the underlying cause of the disease and to identify and test the most promising strategies relevant to Duchenne muscular dystrophy. This holistic approach is truly platform agnostic.

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