Alnylam and Collaborators Publish Research on a new Class of Chemically Modified RNAi Therapeutics

Alnylam Pharmaceuticals, Inc. has announced the publication of a new study in Nature Medicine by scientists from the University of Bonn in collaboration with Alnylam.

The new pioneering research documents the design and evaluation of “3p-siRNAs,” a new class of RNAi therapeutics comprised of small interfering RNAs which are designed and chemically modified to activate RIG-I.

The study showed that a 3p-siRNA specific for the anti-apoptotic factor bcl-2 may be a highly effective anti-cancer agent in several animal models. 3p-siRNAs that are designed toward other gene targets may also have applications in infectious disease and as vaccine adjuvants.

“In our first collaborative paper with the Hartmann lab in 2005, also published in Nature Medicine, we defined certain structural features of siRNAs that were found to stimulate immune responses, ensuring we can reliably avoid these responses in designing our RNAi therapeutics,” said Rachel Meyers, Ph.D., Senior Director of RNAi Lead Development at Alnylam.

Meyers continued, “In this new research, 3p-siRNAs were designed to intentionally promote a potent immune stimulatory mechanism in concert with gene silencing. We believe this approach could represent a potentially new and effective strategy for cancer therapy, as it has been shown that many cancers are better treated through a combination therapy approach.”