Once-in-a-decade breakthrough

Alnylam and Precision NanoSystems form RNAi delivery collaboration

Lloyd Dunlap
CAMBRIDGE, Mass.—Precision NanoSystems Inc. and AlnylamPharmaceuticals Inc. have formed an exclusive collaboration focused on thediscovery and development of novel lipid nanoparticles, known as small lipidnanoparticles (sLNPs), using microfluidics technology.
 
Based on their small particle size of approximately 20nanometers, sLNPs have the potential for broadened biodistribution beyond liverdelivery, a limitation that has long been a challenge in RNA interference(RNAi) therapeutic development. The company declined to answer the question ofhow much broader sLNP-assisted delivery might be in terms of specific organs orsystems.
 
 
Addressing the rationale for the new partnership, Dr.Kenneth Koblan, chief scientific officer at Alnylam, notes that "both companiesrecognized that novel small lipid nanoparticles represent an exciting andinnovative approach in Alnylam's advancement of proprietary LNPs for RNAitherapeutics to significantly improve and broaden biodistribution."
 
"The technology was developed by the University of BritishColumbia, with whom we have collaborated in the past, and is licensedexclusively to Precision NanoSystems for commercialization," Koblan says. "Welook forward to working with Precision NanoSystems to support research effortsaround the discovery of novel sLNPs that we believe have the potential tosignificantly improve and broaden biodistribution." Results from ongoingstudies will inform which disease targets and indications may be best suitedfor this technology, Koblan adds.
 
"Alnylam is leading the translation of RNAi technology intohuman therapeutics, and we look forward to working with them," says Dr. JamesTaylor, CEO of Precision NanoSystems. His company will use its microfluidicstechnology to drive the development of novel lipid nanoparticles.
 
 
Alnylam has long maintained that RNAi promises to be arevolution in biology, representing a breakthrough in understanding how genesare turned on and off in cells, and a completely new approach to drug discoveryand development. The company points out that its discovery has been heralded as"a major scientific breakthrough that happens once every decade or so," andrepresents one of the most promising and rapidly advancing frontiers in biologyand drug discovery today which was awarded the 2006 Nobel Prize for Physiologyor Medicine.
 
RNAi is a natural process of gene silencing that occurs inorganisms ranging from plants to mammals. By harnessing the natural biologicalprocess of RNAi occurring in cells, the creation of a major new class ofmedicines, known as RNAi therapeutics, may be on the horizon. Small interferingRNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam's RNAitherapeutic platform, target the cause of diseases by silencing specific mRNAs,thereby preventing disease-causing proteins from being made. RNAi therapeuticshave the potential to treat disease and help patients in a fundamentally newway, the company states.
 
As part of its "Alnylam 5x15" strategy, the company expectsto have five RNAi therapeutic products for genetically defined diseases inadvanced stages of clinical development by the end of 2015. Three of the fiveprograms have been announced: ALN-TTR for the treatment oftransthyretin-mediated amyloidosis; ALN-PCS for the treatment ofhypercholesterolemia; and ALN-HPN for the treatment of refractory anemia. Thefinal two will be defined later this year.
 
The company has also partnered up in programs in clinical ordevelopment stages, including ALN-RSV01 for the treatment of respiratorysyncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers andALN-HTT for the treatment of Huntington's disease. Among its partners areMerck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin andCubist. Alnylam has also formed Alnylam Biotherapeutics, a division of thecompany focused on the development of RNAi technologies for application inbiologics manufacturing, including recombinant proteins and monoclonalantibodies.

Lloyd Dunlap

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