Tekmira Pharmaceuticals, a biopharmaceutical company specializing in novel RNA interference (RNAi) therapeutics, has announced that Alnylam Pharmaceuticals has reported preliminary results of a Phase I clinical study of an RNAi therapeutic targeting transthyretin (TTR) called ALN-TTR01 for TTR-mediated amyloidosis (ATTR) using the lipid nanoparticle technology (LNP) of Tekmira.
In Tekmira's LNP technology, formerly known as stable nucleic acid-lipid particles or SNALP, RNA molecules are efficiently encapsulated in homogeneous lipid nanoparticles that are efficient in supplying RNAi therapeutics to diseased spots in several preclinical models. The company uses a powerful, expandable and reproducible proprietary method for the production of LNP formulations.
Alnylam Pharmaceuticals has presented the ALN-TTR01 data at the International Symposium on Familial Amyloidotic Polyneuropathy conducted in Kumamoto, Japan, from 20 to 22 November 2011. According to the data, ALN-TTR01 was non-toxic and well administered as well as exhibited quick, dose-dependent, and significantly low levels of serum TTR proteins following the single dosage in ATTR patients.
The President and Chief Executive Officer at Tekmira Pharmaceuticals, Mark J. Murray stated that the latest human clinical trial data is another testament proving that Tekmira's LNP technology allows RNAi activity efficiently and is well administered. The company expects more LNP-enabled clinical information in the following months, including data from TKM-PLK1 program of Tekmira and ALN-PCS program of Alnylam Pharmaceuticals, emphasizing the company’s dominance in the RNAi therapeutic field, he added.