CAMBRIDGE, Mass.—Precision NanoSystems Inc. and Alnylam Pharmaceuticals Inc. have formed an exclusive collaboration focused on the discovery and development of novel lipid nanoparticles, known as small lipid nanoparticles (sLNPs), using microfluidics technology.
Based on their small particle size of approximately 20 nanometers, sLNPs have the potential for broadened biodistribution beyond liver delivery, a limitation that has long been a challenge in RNA interference (RNAi) therapeutic development. The company declined to answer the question of how much broader sLNP-assisted delivery might be in terms of specific organs or systems.
Addressing the rationale for the new partnership, Dr. Kenneth Koblan, chief scientific officer at Alnylam, notes that "both companies recognized that novel small lipid nanoparticles represent an exciting and innovative approach in Alnylam's advancement of proprietary LNPs for RNAi therapeutics to significantly improve and broaden biodistribution."
"The technology was developed by the University of British Columbia, with whom we have collaborated in the past, and is licensed exclusively to Precision NanoSystems for commercialization," Koblan says. "We look forward to working with Precision NanoSystems to support research efforts around the discovery of novel sLNPs that we believe have the potential to significantly improve and broaden biodistribution." Results from ongoing studies will inform which disease targets and indications may be best suited for this technology, Koblan adds.
"Alnylam is leading the translation of RNAi technology into human therapeutics, and we look forward to working with them," says Dr. James Taylor, CEO of Precision NanoSystems. His company will use its microfluidics technology to drive the development of novel lipid nanoparticles.
Alnylam has long maintained that RNAi promises to be a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. The company points out that its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers 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. By harnessing the natural biological process of RNAi occurring in cells, the creation of a major new class of medicines, known as RNAi therapeutics, may be on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by 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, the company states.
As part of its "Alnylam 5x15" 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. Three of the five programs have been announced: ALN-TTR for the treatment of transthyretin-mediated amyloidosis; ALN-PCS for the treatment of hypercholesterolemia; and ALN-HPN for the treatment of refractory anemia. The final two will be defined later this year.
The company has also partnered up in 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. Among its partners are Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin and Cubist. 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.