Alnylam Biotherapeutics presents new data on RNAi technologies at Cell Culture Engineering XII

Alnylam Pharmaceuticals, Inc.May 4 2010

Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the presentation of new data at the Cell Culture Engineering XII conference held in Alberta, Canada from April 25 - 30, 2010. Alnylam Biotherapeutics was formed by Alnylam to develop RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies. The new data presented demonstrated the ability to achieve efficient delivery of siRNAs into Chinese hamster ovary (CHO) cells grown in one liter suspension culture and to achieve potent silencing of CHO host gene targets involved in both cellular apoptotic and metabolic pathways.

“a major scientific breakthrough that happens once every decade or so”

"Our progress to date in our Alnylam Biotherapeutics efforts has been very significant and clearly supports the use of RNAi technologies to potentially transform manufacturing processes for biotherapeutic drugs, including their quality, quantity, and attributes," said Stuart Pollard, Ph.D., Vice President of Scientific and Business Strategy at Alnylam. "These data highlight some of our results in applying RNAi to augment CHO cell growth by targeting both cell death and metabolic pathways that are detrimental to the optimal performance of cells grown in bioreactors."

The new research employed siRNAs designed to target key genes involved in the survival and optimal growth of CHO cells: Bax and Bak, which are apoptotic regulators, and lactate dehydrogenase (LDH), a key metabolic enzyme. siRNAs were delivered to CHO cells, grown in one liter suspension culture using proprietary delivery lipids. Specifically, siRNA-treated cells showed:

• silencing of Bax, Bak, and LDH mRNA levels by approximately 80%;
• greater than 65% decrease in LDH enzyme activity;
• approximately 90% decrease in caspase 3 activity, a well-characterized downstream mediator of apoptotic cell death; and,
• greater than 90% increase of viable cell density.

Alnylam Biotherapeutics expects to continue to advance RNAi technologies for biologics manufacturing, and plans to form partnerships with established biologics manufacturers, selling licenses, products, and services.