Sirna Therapeutics, a leading RNAi therapeutics company, it has signed an exclusive worldwide licensing agreement with the University of Massachusetts Medical School (UMMS) for the rights to patents covering microRNA (miRNA) technology for the modulation of gene expression.

MicroRNA is involved in the RNA interference (RNAi) mechanism and can play a critical role in gene silencing. Blocking the function of miRNAs holds significant potential for the treatment of human disease. In addition to the modulation of gene expression by blocking miRNA function, miRNAs on their own can be used as therapeutic agents.

Like short interfering RNAs (siRNAs), miRNAs are involved in the RNA interference (RNAi) mechanism. However, while siRNAs direct the cleavage of messenger RNA (mRNA) synthesized by a gene, microRNAs appear to predominantly block translation of proteins by binding to the mRNA. The mechanism by which siRNAs and miRNAs induce gene silencing are complementary to one another, thereby presenting a dual approach to harnessing the RNAi mechanism to down regulate pathogenic proteins and viruses.

"Exclusive license to these Zamore miRNA patents, combined with Sirna's existing intellectual property on miRNA, gives our company a leading patent position in the emerging area of miRNA technology and use of miRNA as therapeutic agents or targets," said Bharat Chowrira, VP, Legal Affairs and Chief Patent Counsel. "With these new patents, we have positioned ourselves to capitalize on a broad intellectual property estate, which now enables Sirna to pursue multiple RNAi-based therapeutic approaches," he added.

"This invention by Dr. Zamore and his colleagues represents a powerful approach for modulating miRNA function," James P. McNamara, Executive Director of the Office of Technology Management at the University of Massachusetts Medical School said adding, "We are pleased to license this technology exclusively to Sirna as we believe the company is at the forefront of RNAi-based therapeutic development."

RNA interference (RNAi) is a natural, selective process for turning off genes. RNAi is triggered by short interfering RNA (siRNA) molecules that engage a group of cellular proteins, known as RISC (RNA induced silencing complex). The RISC guides the siRNA to its target messenger RNA (mRNA, the messenger between DNA and proteins) by complementary base pairing for the targeted break-up of the mRNA, thus halting protein expression or viral replication. The RISC-siRNA-complex binds and cleaves multiple mRNA molecules in a catalytic fashion.