The latest in gene technology is being used to offer targeted therapies to patients in order to bring expertise to the area of dystrophy
Mesoderm genetic diseases are rare yet devastating conditions that affect the development of many tissues such as muscle, connective tissue, cartilage and bone. Duchenne Muscular Dystrophy (DMD) is such a disease with a high level of unmet clinical need.
It is an X chromosome-linked genetic disease, characterised by progressive deterioration of skeletal and cardiac muscle, leading to a variable but progressive limitation of the patient’s mobility, including confinement to a wheelchair and heart and/or respiratory failure, typically by 30 years of age.
DMD is caused by a range of mutations in the dystrophin gene, it is incurable and does not yet have a therapy with long lasting or curative benefits. Care for DMD patients is intensive, time consuming and expensive with an associated cost of up to £65,000 per year of a patient’s life. The potential market size for therapies is estimated to be in the region of $1bn and the technology also offers possibilities to expand our approach to other diseases.
The University of Manchester are developing a safe, “immune privileged” mesoderm derived progenitor cell line which will be applied in cell-mediated gene therapies. This enabling platform technology will be applicable to the correction of many rare genetic diseases affecting the mesoderm. Disease-specific therapeutic cells are derived by genetic modification of the immune privileged cells to include a therapeutic payload, which should provide both efficient engraftment and long-lasting therapeutic effect.
The team are currently conducting a Proof-of-Concept study targeting their universal mesoderm cell to a specific DMD mutation, leveraging the team’s expertise and know-how in clinical application of cell therapies to DMD. They expect that the combination of their “immune-privileged cell” and correcting elements of the dysfunctional endogenous dystrophin gene will provide for efficacious treatment with a good cost/benefit profile