Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Alnylam Pharmaceuticals, an RNAi therapeutics company, has published its pre-clinical research in the journal Molecular Therapy, revealing key mechanisms related to the systemic delivery of RNAi therapeutics using lipid nanoparticles (LNPs).
Subscribe to our email newsletter
According to the Alnylam, the new study performed in collaboration with scientists at the Max Planck Institute of Molecular Cell Biology and Genetics and AlCana Technologies, described a mechanism for endogenous apolipoprotein E (apoE)-mediated targeting of LNPs to the liver, demonstrates alternative ligand-directed targeting strategies for liver delivery of RNAi therapeutics, and potential targeting approaches for delivery to tissues and cell types beyond the liver.
Preliminary results from this study were previously presented at the Advances in Biopharmaceuticals Keystone Symposium in January 2010, demonstrating that ApoE is an endogenous targeting ligand for neutrally charged ionizable LNPs (iLNPs), but not certain cationic LNPs (cLNPs).
Akin Akinc, associate director of research at Alnylam, said: “In recent months, we have made tremendous progress in the delivery of RNAi therapeutics, a critical determinant for advancement of this promising new class of medicines to patients and for realizing the fullest potential of this technology.
“In particular, the findings published reveal key mechanisms for the delivery of LNP-encapsulated siRNAs to the liver, and more generally, suggest strategies for achieving targeted delivery to tissues and cell types beyond the liver. These mechanistic findings, along with our recent work on the discovery of next-generation LNPs, demonstrate the considerable progress made both in our understanding and in our utilization of LNPs for the delivery of RNAi therapeutics.”