RNAi positively propels Alnylam cancer vaccine in preclinical study

Alnylam Pharmaceuticals ($ALNY) and the company's Dutch research partners say they've reached a big milestone using RNAi to drastically boost the potential of a cancer vaccine under development to generate an immune response.

Scientists at Radboud University Nijmegen Medical Centre in the Netherlands also participated in the research, which is detailed in the journal Cancer Immunology Immunotherapy.

The dendritic cell cancer vaccine showed promising results targeting PD-L1 and PD-L2 proteins, the researchers said. Both proteins block the strong T-cell response needed to fight the tumors, they explain, so disabling them potentially boosts the effectiveness of the cancer vaccine.

Rachel Meyers, Alnylam's vice president of research and RNAi lead development, said in a statement that the finding shows "the potential of an ex vivo RNAi approach" for dendritic cell vaccines to fight tumors is real, and that "this new strategy could significantly enhance the potency of cellular immune therapy in cancer." The vaccine used lipid nanoparticle-formulated siRNA--nanoparticles are thought to be a better way to deliver RNAi to its target, so it can effectively turn off bad genes that cause disease.

RNAi drug development remains a struggle, and Alnylam slashed a third of its jobs earlier this year in order to focus more on programs such as new drugs to treat hemophilia and transthyretin-mediated amyloidosis. This has been an encouraging summer for the company at the same time. In July, the company touted positive clinical results for ALN-AT3, a new RNAi hemophilia drug.

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