Scleroderma Patent

 NovaGenetix represents since some years a patent for Scleroderma treatment hold by the University of Zurich. This patent may create some interest to develop an ointment on the basis of miR-29, which would be a novelty. The patent is maintained in most European countries, the United Kingdom, Switzerland, Australia and the United States.

 Scleroderma is a chronic autoimmune disease characterized by a fibrosis of the skin and internal organs. The high morbidity and mortality is the consequence of the failure of affected organs such as the lungs. In the United States alone approximately 300.000 people are affected. To date there is no therapy available to slow or prevent the disease progression. Therefore, there is a substantial need for a specific anti-fibrotic therapy. miR-29 is a small non-coding micro RNA that is involved in regulating gene expression.

This invention relates to a novel pharmacological treatment of scleroderma comprising the administration of miR-29. miR-29 was found to be strongly downregulated in Scleroderma fibroblasts as well as skin biopsies from Scleroderma patients compared to healthy controls. Furthermore the enforced expression of miR-29 in Scleroderma skin fibroblasts resulted in downregulation of collagen on mRNA and on protein level. These experiments provide robust evidence that the miR-29 family negatively regulates major pathogenic pathways in Scleroderma.

Three publications are recommended for further studying and reviewing:

• Armando Gabrielli et al: Scleroderma, New England J. of Medicine 360, 1989 (2009)
• Simon Strietholt, Steffen Gay et al: Epigenetic modifications in rheumatoid arthritis, Arthritis Research & Therapy 10, 219 (2008)
• Britta Maurer, Steffen Gay et al: MicroRNA-29, a key regulator of collagen expression in systemic sclerosis, Arthritis & Rheumatism 62, 1733 (2010)