“The Duchenne Research Fund is very excited to support these innovative projects with Solid Biosciences as they are addressing some of the major challenges in DMD research,” said Ronald Cohn, MD, FACMG, head of the DRF’s Scientific Advisory Board. “Treatments that target fibrosis will be necessary to maintain or even reverse functional deficits in boys with DMD. In addition, the development of novel biomarkers to help better inform the design and execution of clinical trials has the potential to revolutionize how we analyze any future therapies for this disease.”
Solid added the LTBP4 program to its research pipeline in 2016, a collaboration with Dr. Beth McNally’s lab at Northwestern University, to better understand the compound’s potential to reduce signs of muscle disease and increase overall function. It is believed that the protein LTBP4 prevents the activation of a key biological pathway (called TGF-beta), an important factor in inflammation and fibrosis. In a natural history study of Duchenne patients, the presence of a modified version of this LTBP4 protein correlated with patients’ ability to walk until later ages. If successful, this approach may be complimentary therapy to our Gene therapy treatment.
Solid is also exploring novel less invasive biomarkers, which aim to advance the entire field of drug development for Duchenne. This work includes non-invasive imaging, activity monitoring and blood-based biomarkers. These efforts aim to enable a greater population of non-ambulatory patients to participate in clinical trials, minimize the physical and emotional toll of invasive biopsies, and more sensitively capture functional changes.
“In this industry, we have learned that coming together to form alliances can change history,” said Gilad Hayeem, president of Solid Biosciences. “The DRF’s sophisticated and well-informed leadership recognizes the challenging landscape for drug approval. Through their commitment to help fund Solid’s biomarker and LTBP4 compound work, we hope we will advance the entire field of drug development for Duchenne.”