Session Speaker

Type I Collagen Antisense Oligonucleotides for the Treatment of Fibrotic Diseases
Rahul K. Nath, Weijun Xiong, Chandra Somasundaram, Jessica Li, Ka Bian and Ferid Murad
USA

Fibrotic diseases are chronic diseases that are currently untreatable effectively. Many may become fatal and most cause significant morbidity. They are characterized by progressive fibrosis associated with excessive collagen synthesis and accumulation, resulting in functional impairment of affected organs. Currently, there is no cure for these diseases. Hence, there is a critical need for an effective and safe therapy. The objective of our research is to bring our patented drug, type I collagen antisense oligo-nucleotides (AS60-ODN), to clinical trial for the treatment of various fibrotic diseases. We have previously reported that AS60-ODN reduced the type I collagen expression in a novel ex vivo human skin wound model. Recently, we have tested the effect of AS61-ODN (mouse analog of human AS60-ODN) in bleomycin induced pulmonary fibrosis in mice. AS61-ODN significantly reduced the mRNA and protein expression of type 1 collagen by 45.2 % (p < 0.015) and 35.0 % (p < 0.004), and there was less disruption of the lung architecture 21 days after bleomycin instillation. We have also evaluated AS60-ODN for acute and chronic toxicities. Further, we are working to get IND approval from the FDA, to take AS60-ODN to clinical trial, and eventually to treat patients with fibrotic diseases.