U.S. Patent 7,641,915, awarded to Sirna Therapeutics, Inc (San Francisco, CA), details novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. Sirna lipid nanoparticle compositions can be used for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism.
Sirna inventors TongQian Chen, Chandra Vargeese, Kurt Vagle, WeiMin Wang and Ye Zhang created novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules.
Molecules which can be delivered include antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, dsRNA, allozymes, aptamers, decoys and their analogs.
Other deliverable molecules include small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules, to relevant cells and/or tissues, such as in a subject or organism.
Sirna's novel cationic lipids, microparticles, nanoparticles and transfection agents are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism. The patented Sirna compositions are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).
The scientific community considers RNA interference the breakthrough biological discovery of the decade with the potential to change how diseases are treated. Sirna Therapeutics is at the forefront of the effort to create RNAi- based therapies and leverage the vast potential of this technology to ultimately treat patients.
