This New Collaboration Aims to Create Gene Therapies for Duchenne Muscular Dystrophy

According to CureDuchenne, two companies have began a collaboration in an effort to create new gene therapies for Duchenne muscular dystrophy (DMD). Ultragenyx and Solid Bioscience are both using their latest developments, a differentiated microdystrophin construct and HeLa producer cell line (PCL) manufacturing platform, to make the best possible treatment for those with DMD.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is one of the nine forms of muscular dystrophy. Affected individuals experience progressive muscle weakness and wasting due to a lack of dystrophin. Symptoms include issues with motor skills, muscle weakness, falling, fatigue, issues with changing positions, problems walking, learning disabilities, and eventually heart disease and respiratory failure. All of these effects are caused by a mutation passed down on the X chromosome in a recessive pattern. There is currently no cure for this condition, but treatments can help to manage symptoms.

About the Collaboration

The goal of this collaboration is to create a new gene therapy for DMD. In order to do so, both Ultragenyx and Solid Biosciences will bring their own respective expertise and technology.

Ultragenyx enters the agreement with its HeLa producer cell line (PCL) manufacturing platform, which brings a 2,000 liter AAV-based gene therapy product manufacturing that is available on a commercial scale. It has the ability to mass produce the therapy for a smaller price, making it highly advantageous. This company also has experience and expertise in developing treatments for rare diseases.

On the other side of the agreement, Solid Biosciences has a proprietary microdystrophin construct that is already showing benefits for DMD in preclinical models. Mouse models were able to better resist fatigue when given the construct. The first human trial has shown positive results as well, and researchers are looking forward to the next steps. In addition to this construct, Solid will provide their extensive knowledge and expertise on muscle biology.

The terms of the collaboration, Ultragenyx contributed $40 million to Solid in investments and provide further funds when milestones are made in development. In return, Ultragenyx has exclusive licensing rights to not only the gene therapy currently in development, but any other product that utilizes the proprietary microdystrophin construct. Hopefully the collaboration goes well, as it could result in a viable treatment option for those with DMD.

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