Alnylam Pharmaceuticals, a leading RNAi therapeutics company announced that it has consolidated the key intellectual property (IP) for RNA activation (RNAa), creating a dominant IP position in this new area of biology.

RNAa is an emerging biological discovery involving double-stranded RNAs that target promoter regions in chromosomal DNA resulting in transcriptional activation of genes. The transcriptional activation, or up-regulation, of genes results in an increase in mRNA and protein production.

Accordingly, this technology may have applications in a range of human disorders such as certain genetic diseases and cancer where the aberrant low expression of certain proteins is known to occur. In order to consolidate IP in this field, Alnylam has completed exclusive license agreements with the leading academic institutions working in this area: the Corey lab at the University of Texas Southwestern Medical Center (UT Southwestern); the Li lab at the University of California San Francisco (UCSF); and the Gage lab at the Salk Institute for Biological Studies (Salk).

"Alnylam is committed to scientific and IP leadership across all areas of modern biology's RNA revolution, including our ongoing efforts in RNAi and microRNA therapeutics, and now RNAa," said John Maraganore, PhD, chief executive officer of Alnylam. "While there's more to understand in this emerging biology, RNAa defines a new application for double-stranded RNAs that could have the potential to create an entirely new therapeutic platform for Alnylam. Many human diseases are caused by the abnormally low expression of proteins, and RNAa could be used to treat these disorders through selective transcriptional gene activation. Obtaining exclusive access to these key RNAa patents positions Alnylam to lead in the translational research of this biology as it advances toward in vivo validation and provides a solid foundation for business execution, as we've demonstrated in the past with our RNAi and microRNA platforms."

"We're excited to be working with Alnylam, as they have demonstrated a commitment to scientific excellence in their publications and academic collaborations," said David Corey, PhD, Professor of Pharmacology and Biochemistry at UT Southwestern. "We look forward to collaborating on the emerging science of RNAa biology to further explore the breadth of applications for transcriptional gene activation with agRNAs."

RNAa is mediated by double-stranded RNAs, possibly including endogenous miRNAs that target the promoter regions of genes in chromosomal DNA. So-called "anti-gene RNAs," or agRNAs, appear to increase transcription by complementary base-pairing with corresponding promoter sequences which enhance recruitment of the cellular RNA polymerase II and therefore increase the target gene's expression. In vitro studies, agRNAs can increase the levels of a target gene's mRNA by nearly 10-fold, resulting in an increased level of the target gene's protein.

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development.

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is applying its therapeutic expertise in RNAi to address significant medical needs, many of which cannot effectively be addressed with small molecules or antibodies, the current major classes of drugs.