Alnylam presents data on RNAi technologies at MIT Center Summit

Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, presented new data at the MIT Center for Biomedical Innovation's Biomanufacturing Summit from its platform efforts aimed at developing RNAi technologies for applications in biologics manufacturing. The new studies show that siRNAs designed to target a virus known to infect CHO cells - cells that are commonly used to produce recombinant proteins and monoclonal antibodies - can potently block infection and viral replication. RNAi technologies to prevent or treat viral infection of manufacturing cell lines could provide a novel strategy to ensure product quality of biotherapeutic products for patients.

“By all accounts, these encouraging new findings are representative of the rapid progress we are making in this overall effort, and highlight important new applications of our biotherapeutics platform.”

"We remain excited about our Alnylam Biotherapeutics platform and how this application of RNAi technology has the potential to transform the manufacturing of biotherapeutic products. These new data describe applications of RNAi technologies to block viral infection of CHO cells - an approach that could preserve quality in biologics manufacturing and also maintain consistency of product supply," said Ken Koblan, Ph.D., Chief Scientific Officer at Alnylam. "By all accounts, these encouraging new findings are representative of the rapid progress we are making in this overall effort, and highlight important new applications of our biotherapeutics platform."

In the new study, performed in collaboration with Dr. Ian Goodfellow from Imperial College London, siRNAs were designed and synthesized toward vesivirus, a virus known to infect CHO cells that can result in adverse effects on product quality and/or potential interruption in biologics manufacturing and product supply. Vesivirus is a small RNA virus in the calciviridae family that can be found in animal-derived products used to establish manufacturing cell banks and/or used during the course of manufacturing processes. siRNAs were designed toward the helicase and polymerase genes of vesivirus (strain 2117) and were screened for in vitro antiviral activity, resulting in identification of nine candidate siRNAs for further evaluation. In a treatment protocol, CHO cells grown in suspension culture were infected with vesivirus and then treated with anti-vesivirus siRNAs. The siRNA treatment was found to potently block viral replication with an approximate 2-log reduction in vesivirus RNA, as measured by PCR. In aggregate, these results provide key proof of concept for applications of RNAi technologies as an anti-viral strategy in biologics manufacturing that could also apply to other endogenous and exogenous viral cell culture pathogens.

Source: Alnylam Biotherapeutics