Alnylam Pharmaceuticals, a Cambridge, MA company focused on RNAi-based therapeutics, has filed an application to initiate Phase I trials for ALN-PCS, its second-generation lipid-nanoparticle delivery technology to treat severe hypercholesterolemia, or high cholesterol in the blood.
"We are excited about the potential for ALN-PCS to make an impact in the treatment of severe hypercholesterolemia as this RNAi therapeutic targets both intracellular and extracellular PCSK9, a target validated by human genetics that is known to play a central role in LDL cholesterol metabolism," Alnylam's Akshay Vaishnaw said in a release.
The trial is part of what the company calls its "Alnylam 5x15" strategy, which "establishes a path for development and commercialization of novel RNAi therapeutics to address genetically defined diseases with high unmet medical need." By the end of 2015, the company expects to have five such RNAi therapeutic programs in advanced clinical development.
Back in March of this year, Tekmira Pharmaceuticals filed litigation against Alnylam concerning its lipid nanoparticle delivery technology.
- read the release
- and a blurb in Mass High Tech