Isis-Rosetta Collaborate to Develop Micro-RNA Related Therapies
News Feb 23, 2006
Isis Pharmaceuticals, Inc. and Rosetta Genomics Ltd. have announced a joint research collaboration to discover and develop antisense drugs that regulate microRNAs for the treatment of prevalent type of liver cancer, hepatocellular carcinoma (HCC).
The collaboration will bring together the complementary expertise of both companies in miRNA and leverage Rosetta's database of miRNA genes and Isis' expertise in oligonucleotide chemistry and antisense drug discovery and development.
Antisense drugs bind to complementary RNA sequences, such as miRNAs, inhibiting the function of miRNAs.
Research recently conducted by Isis has shown that antisense inhibition is a powerful technique to regulate the function of miRNAs, which are naturally expressed small RNAs believed to play a central role in the progression of many forms of cancer and other serious illnesses.
"We believe miRNAs are most easily accessible using antisense technologies and look forward to working with Rosetta Genomics to discover antisense drugs targeted to miRNAs for the treatment of liver cancer," said C. Frank Bennett, Ph.D., Senior Vice President, Antisense Research at Isis.
"Our recently published research demonstrates pharmacological activity in vivo using well validated antisense chemistry to inhibit miRNAs."
"We believe our collaborative efforts with Rosetta Genomics will enable both companies to take advantage of miRNAs as potential new drug targets for the treatment of a range of diseases, including liver cancer."
Isis recently published new research in the February 2006 issue of Cell Metabolism demonstrating that antisense inhibition is a powerful technique in regulating the function of miRNAs in the liver.
To determine the role of miR-122 in the adult liver, Isis scientists inhibited miR-122 with an antisense oligonucleotide in mice.
The antisense inhibition of miR-122 in normal and high fat-fed mice resulted in a significant improvement in numerous metabolic and cardiovascular risk factors as evidenced by reduced plasma cholesterol levels, increased hepatic fatty-acid oxidation, decreased hepatic fatty-acid and cholesterol synthesis rates and reduced fat in the liver (steatosis).
These results implicate miR-122 as a key regulator of cholesterol and fatty-acid metabolism in the adult liver and suggest that miR-122 may be an attractive therapeutic target for cardiovascular and metabolic diseases.
"MiRNAs appear to regulate at least one-third of all gene expression and represent a new class of drug targets for the pharmaceutical industry," said Amir Avniel, President of Rosetta Genomics.
"We are pleased to join forces with Isis and view antisense as the optimal platform for inhibiting miRNA function."
"Given the prevalence of HCC and the limited treatment options for patients with hepatocellular carcinoma, we are especially eager to begin working with Isis to discover and develop potential new treatment options for patients around the world."
As genome editing technologies advance toward clinical therapies, they are raising hopes of a completely new way to treat disease. However, challenges need to be addressed before potential treatments can be widely used in patients. To tackle these challenges, the National Institutes of Health has launched the Somatic Cell Genome Editing program, which has awarded multiple grants including more than $3.6 million to assess the safety of genome editing in human cells and tissues.