Alnylam Reports Positive Results for Nanotech-Enabled RNAi Therapeutic Targeting PCSK9

Tekmira Pharmaceuticals, a company specializing in novel RNA interference (RNAi) therapeutics, has announced that Alnylam Pharmaceuticals has reported encouraging initial results from its continuing human clinical study of a target-specific RNAi therapeutic for PCSK9 called ALN-PCS for treatment of dangerous hypercholesterolemia using the lipid nanoparticle technology of Tekmira.

According to the report of Alnylam Pharmaceuticals, ALN-PCS was well tolerated and safe to use. Moreover, ALN-PCS showed up to 66% of RNAi silencing of PCSK9, a value that is statistically important. It also reduced the levels of bad cholesterol or low-density lipoprotein cholesterol by up to 50%, a clinically significant value.

The President and Chief Executive Officer at Tekmira Pharmaceuticals, Dr. Mark J. Murray stated that the company is happy about the positive results from another clinical study, which corroborates that the company’s lipid nanoparticle technology allows RNAi activity and is well tolerated. These results further extend the company’s dominance in the RNAi therapeutic field, he added.

Murray further said that the recent IND approval for Tekmira’s TKM-Ebola product and the positive results from the clinical study of ALN-TTR01, and the latest encouraging data from the Phase I trial of ALN-PCS are in line with the company’s expectations on the RNAi field. The company can now refer to several clinical studies in different clinical indications to generate results in order to assess the clinical efficacy, tolerability and safety of the RNAi therapeutics based on the lipid nanoparticle technology, he concluded.

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