Alnylam Pharmaceuticals, Inc., a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi, has earned a development milestone payment from GlaxoSmithKline (GSK), totalling $3.2 million. The milestone payment is related to progress in the companies’ collaboration on the use of VaxiRNA technology for the production of GSK influenza vaccine. Alnylam’s VaxiRNA platform uses RNAi technology for the enhanced manufacturing of vaccine products.

“We have made important progress with GSK in advancing Alnylam’s VaxiRNA technology, and are pleased to have achieved a key milestone in our collaborative effort,” said Rachel Meyers, PhD, vice president, Research and RNAi Lead Development. “At Alnylam, we continue to pioneer innovative applications of our RNAi platform that could have a transformative impact in medicine and the biopharmaceutical industry at large. Our VaxiRNA platform is representative of this strategy, where RNAi technology is being used to increase virus titers in the production of vaccine products. With GSK, we are applying RNAi technology with the aim to enhance influenza vaccine production in cell culture. We look forward to continuing our efforts in this strategic collaboration.”

Alnylam’s VaxiRNA platform applies siRNA for the silencing of specific genes that limit or prevent efficient manufacturing of vaccine antigens, including those produced in cell culture and in eggs. The VaxiRNA platform stems from work Alnylam has performed as part of its Alnylam Biotherapeutics efforts, where RNAi technology has been applied to improve the manufacturing of biotherapeutic products, such as recombinant proteins and monoclonal antibodies. Alnylam and GSK formed their collaboration in the fourth quarter of 2011. The effort is focused initially on influenza vaccine production in cell culture systems. Under the terms of the agreement, GSK will provide research funding and certain milestone payments to Alnylam, such as the $3.2 million received this quarter. If successfully applied in the manufacturing of commercial product, Alnylam will receive payments on unit product sales of commercialized vaccine product. In addition, GSK has obtained an option for VaxiRNA applications toward two additional vaccine products.

Alnylam is applying RNAi technology to improve the manufacturing processes for vaccines in an effort called VaxiRNA. With VaxiRNA, Alnylam is using siRNA that silence specific genes in vaccine production systems, such as cells or eggs, which limit or prevent the efficient manufacture of vaccine products. New innovations in vaccine manufacturing are needed to enable the scale and speed of global immunization for a number of pathogens. In 2011, Alnylam formed a VaxiRNA partnership with GlaxoSmithKline for influenza vaccine production.

RNAi (RNA interference) represents a breakthrough in understanding how genes are turned on and off in cells, and adds a new approach to drug discovery and development. Its discovery represents a promising and rapidly advancing frontier in biology and drug discovery today, which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. This process can be used to enhance the manufacturing of vaccines or medicines. In addition, by harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics might be possible in the future. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.