Precision NanoSystems and Alnylam Pharmaceuticals formed an exclusive collaboration focused on the discovery and development of 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, the firms point out.
Alnylam’s core focus is on RNAi therapeutics for the treatment of genetically defined diseases including ALN-TTR for the treatment of transthyretin-mediated amyloidosis, ALN-PCS for the treatment of severe hypercholesterolemia, and ALN-HPN for the treatment of refractory anemia. Of these, ALN-TTR is the most advanced, having reached Phase I trials. The company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015 under its “Alnylam 5x15™” strategy.
Alnylam has additional partner-based programs at various stages of development. ALN-RSV01 for the treatment of respiratory syncytial virus infection is in Phase II studies. Kyowa Kirin owns Asian rights, and Cubist has licenses covering the rest of the world. ALN-VSP for the treatment of liver cancer is reportedly ready to enter Phase II, and Alnylam is looking for a partner. ALN-HTT for the treatment of Huntington disease is being developed by Medtronic and the CHDI Foundation.
Alnylam’s RNAi intellectual property has enabled it to form alliances with a range of companies including Biogen Idec, Merck & Co., Novartis, Roche, and Takeda. In addition, Alnylam and Isis co-founded Regulus Therapeutics to focus on miRNA therapeutics. Regulus has formed partnerships with GlaxoSmithKline and sanofi-aventis. Alnylam also set up Alnylam Biotherapeutics as a division of the company to concentrate on the development of RNAi technologies for application in biologics manufacturing.