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이번주 목요일(10월 29일) 세미나가 진행됩니다.               

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감사합니다.  

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제  목 :  Therapeutic siRNAs for Human Disease

연  사 : Muthiah Manoharan, Ph.D (Alnylam Pharmaceuticals)

일  시 : 2015년 10월 29일 (목) 오후 4시 30분 

장  소 : 화학관 세미나실 (330226호실)

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Therapeutic siRNAs for Human Disease

                         

Synthetic small interfering RNAs (siRNAs) act as therapeutic agents through the RNA interference (RNAi) pathway and are specific and potent inhibitors of gene expression. These agents may be designed to target disease pathways previously considered “undruggable”. Numerous proof-of-concept studies both in animal models of human disease and in clinical trials demonstrate the broad potential and therapeutic value of RNAi therapeutics. The major challenge for the successful development of systemically delivered RNAi therapeutics had been the efficient delivery into organs or tissues and cells of interest to elicit RNAi mediated knockdown of faulty genes and effective translation of these approaches into clinic.   At Alnylam Pharmaceuticals, we have developed chemical modifications of siRNAs to provide them with “drug-like” properties. Furthermore, efficient liver delivery approaches have been developed  and clinical trials are advancing with RNAi therapeutics formulated in lipid nanoparticles (LNPs) for intravenous administration. More recently, systemic delivery of therapeutic siRNAs to liver hepatocytes by subcutaneous administration has been achieved by conjugating chemically modified siRNAs with multivalent N-acetylgalactosamine (GalNAc) residues that are recognized by the asialoglycoprotein receptor (ASGPR). siRNA-GalNAc conjugates efficiently target and silence disease-causing genes produced in liver hepatocytes. Using this conjugation platform, Alnylam is advancing several RNAi agents specific for liver targets through pre-clinical and clinical development to address diseases with highly unmet medical need. Our progress with the chemistry of siRNAs, their conjugates and applications in several therapeutic areas will be presented.