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 has presented positive preclinical data from its hypercholesterolemia program in collaboration with UT Southwestern Medical Center in Dallas for reducing bad cholesterol levels.
Subscribe to our email newsletter
The program used RNAi therapeutics directed to the disease target proprotein convertase subtilisn/kexin type 9.
The data shows that in vivo systemic administration of an RNAi therapeutic in non-human primates resulted in efficient silencing of the PCSK9 gene as measured by circulating PCSK9 plasma levels which were reduced by up to 70% of pre-dose levels. RNAi-mediated gene silencing was associated with rapid reductions in LDL cholesterol (bad cholesterol) levels by 40 to 60% of pre-dose levels.
Victor Kotelianski, vice president for research at Alnylam, said: “We are very excited about the significance of these findings, which show for the first time a strong effect for acutely and durably improving cholesterol levels by antagonizing PCSK9 with an RNAi therapeutic in non-human primates. Although PCSK9 is well validated based on human genetics, it has been a difficult protein to target using traditional drug discovery approaches.”
Jay Horton, Professor of Internal Medicine and Molecular Genetics, UT Southwestern Medical Center at Dallas, said: “Based on its novel mechanism of action and preclinical data to date, an RNAi therapeutic targeting PCSK9 has the potential to lower LDL cholesterol, while possibly functioning synergistically with statins in the treatment of hypercholesterolemia.”