TVN-102 is an experimental treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). It is being developed by Tivorsan Pharmaceuticals and was granted orphan drug status by the U.S. Food and Drug Administration (FDA) in 2016.
How TVN-102 works
DMD and BMD are heritable muscle disorders caused by mutations in the gene that provides instructions for the production of the dystrophin protein, which gives structure and stability to muscle fibers. When there is not enough functional dystrophin, skeletal and heart muscles weaken and waste away.
Biglycan is a muscle protein that activates a compensatory pathway that can potentially overcome the damaging effects of dystrophin loss in muscular dystrophy patients. Biglycan increases the expression of utrophin, a protein similar to dystrophin that can partially compensate for the absence of dystrophin.
Biglycan is widely expressed in the body as a proteoglycan, a protein that is chemically modified with a sugar molecule called glycosaminoglycan (GAG). This alters the function of the protein. In muscle, biglycan is also found in a non-glycanated form. Non-glycanated biglycan is the only form that is efficient in muscular dystrophy.
TVN-102 is a recombinant, non-glycanated biglycan that would be delivered systemically by injection into the bloodstream, and is predicted to increase the production of utrophin in muscles, strengthening the muscle fibers and preventing or reversing muscle wasting.
TVN-102 in clinical trials
Although clinical trials are currently planned, no information is available yet on how they will be structured.
Preclinical studies in dystrophin-deficient mice, published in the journal Matrix Biology, have demonstrated that systemically administered recombinant biglycan improves the health, structure, and function of muscles with no signs of toxicity.
An additional study, published in Human Gene Therapy, used dystrophin-deficient mice but delivered recombinant biglycan as gene therapy. Mice were injected with a modified virus that, rather than causing disease, inserts a healthy copy of the gene encoding for biglycan into cells, allowing them to produce more biglycan. The mice showed increased expression of biglycan and utrophin as well as improved muscle fiber connections after treatment.
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