GSK to Use Alnylam Pharmaceuticals' RNAi Technology for Vaccine Production

Alnylam Pharmaceuticals, Inc. recently announced it has formed a collaboration with GSK focused on Alnylam’s VaxiRNA technology for certain GSK vaccine products, including influenza. VaxiRNA is a new RNAi technology for the enhanced production of viruses used in the manufacture of vaccine products. GSK, a leading vaccine manufacturer, is the first company to form a collaboration accessing this new platform.

“At Alnylam, we continue to pioneer innovative opportunities for our RNAi platform that could have transformative impact in medicine and the biopharmaceutical industry. This is exemplified with VaxiRNA, where siRNA technology is being used to increase virus titers in the production of vaccine products. Because production of vaccines can be limiting for the scale and speed of global immunization against many pathogens, including influenza, we believe VaxiRNA can become an enabling technology for the broader vaccine industry,” said John Maraganore, PhD, CEO of Alnylam. “As we advance our VaxiRNA effort, we are excited to form a new collaboration with GSK, a global leader and innovator in vaccine development and manufacturing. With GSK, we aim to apply VaxiRNA technology to enhance influenza vaccine production in cell culture, where Alnylam will receive research funding, in addition to potential milestones and payments on unit sales of commercialized vaccine product.”

Alnylam’s VaxiRNA platform applies siRNAs for the silencing of specific genes that limit or prevent efficient growth of viruses in vaccine manufacturing systems, including those in cell culture and also 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 manufacture of biotherapeutic products, such as recombinant proteins and monoclonal antibodies. This new platform addresses the significant unmet commercial need for innovative technologies that can improve the manufacture of vaccine products, especially where vaccine production is a limiting factor for the scale and speed of global immunization needs.

The GSK collaboration is focused initially on influenza vaccine production in cell culture systems. Under the terms of the agreement, GSK will provide funding and certain milestone payments to Alnylam. If successfully applied in the manufacture of commercial product, Alnylam will receive payments on unit product sales. In addition, GSK has obtained an option for VaxiRNA applications toward two additional vaccine products.

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-APC for the treatment of hemophilia. As part of its Alnylam 5×15 strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015.

Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies, including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies.