Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the presentation of new data at the IBC 6th Annual Cell Line Development and Engineering conference held in San Francisco, California from June 21 - 24, 2010. Alnylam established Alnylam Biotherapeutics to focus on the application of RNAi technology in manufacturing processes for biotherapeutics. The new results describe the discovery of highly effective novel delivery lipids (NDLs) that efficiently deliver siRNAs to manufacturing cell lines with no measurable adverse effects on cell density or viability. Furthermore, the NDLs demonstrate durable target gene silencing following a single dose, improved protein quality, and scalability to at least 40 liters.
“a major scientific breakthrough that happens once every decade or so”
"The new research presented describes the discovery of proprietary delivery lipids that are superior to commercially available lipids in their cell tolerability and efficacy. Importantly, we have now demonstrated the application of RNAi technology with CHO cells grown in 40 liter bioreactors, with effects comparable to what we observed in one and three liter cell cultures," said Stuart Pollard, Ph.D., Vice President, Scientific and Business Strategy of Alnylam. "Our efforts in Alnylam Biotherapeutics are continuing to advance at a very rapid pace, clearly demonstrating the potential of RNAi technology to broadly and significantly improve the quality, quantity, and attributes of biotherapeutics."
Studies were performed using Alnylam's proprietary NDLs, compared with commercially available lipids, to deliver siRNAs into Chinese Hamster Ovary (CHO) cells. Results showed:
- improved tolerability for NDLs compared with commercially available lipids, with no measurable adverse effects on CHO cell growth rates or cell viability;
- efficacy of target gene silencing, specifically a greater than 80% reduction in CHO cell lactate dehydrogenase activity, following a single dose administration of an siRNA with effects lasting over one week;
- scalability of RNAi technology with comparable target gene silencing activity in CHO cell cultures grown in one, three, and 40 liter bioreactors; and,
- ability to alter attributes of a recombinant monoclonal antibody, specifically a greater than 85% reduction in fucosylation, by targeting CHO cell glycosylation pathways.
Alnylam Biotherapeutics is advancing RNAi technologies to improve the quantity and quality of biologics manufacturing processes in mammalian cell culture, such as CHO cells, with the aim of transforming the $100 billion biologics marketplace comprised of recombinant proteins and monoclonal antibodies. In this application, RNAi technology can be applied to the improvement of manufacturing processes for existing marketed drugs, new drugs in development, and for the emerging biosimilars market. Alnylam has developed proprietary delivery lipids that enable efficient delivery of siRNAs into CHO cells as well as other cell systems that are used for the manufacture of biologics.
As Alnylam Biotherapeutics advances the technology, it plans to form partnerships with established biologics manufacturers, selling licenses, products, and services.