Inspired by tiny particles that carry cholesterol through the body, MIT chemical engineers have designed nanoparticles that can deliver snippets of genetic material that turn off disease-causing genes. This approach, known as RNA ...
Tags: RNA, MIT, Gene Silencing, Nanoparticle
Alnylam Pharmaceuticals, a RNAi therapeutics company, announced that the US Food and Drug Administration (FDA) has granted fast track designation to patisiran (ALN-TTR02) for the treatment of transthyretin (TTR)-familial amyloid ...
Tags: Alnylam, Fast Track Designation
RNAi therapeutics company Alnylam Pharmaceuticals has obtained additional orphan drug designation from US FDA for a RNAi therapeutic, ALN-AT3, to treat hemophilia A. With the additional approval, orphan drug designation for GalNAc ...
Tags: Alnylam, Orphan Drug
Alnylam Pharmaceuticals and Ascletis Pharmaceuticals have entered into a partnership to develop a systemically delivered RNAi therapeutic, ALN-VSP, for the treatment of liver cancers including hepatocellular carcinoma (HCC). Under the ...
Tags: Alnylam Pharmaceuticals, Ascletis Pharmaceuticals, HCC, ALN-VSP