Leaders in Pharmaceutical Business Intelligence (LPBI) Group

LIVE from Kresge Auditorium @MIT

“There’s no doubt that RNA has therapeutic potential; the question is ‘how do you get it where it needs to be’?” Dan Anderson 

Key concepts captured:

• Chemical modifiers
• sequence selection
• materials to be used for RNA as a drug:
• Combinatorial synthesis of lipid-like material – complex them with RNA nanoparticles
• In vivo screening for delivery to liver: Lipidoid/Factor siRNA
• TTR – structure/function studies
• Next generation LNPs – Potency Improvements with novel Lipids
• One injection – 10 genes – knockout all 10: Inhibition of 10 genes
• siRNA delivery is NOT limited to hepatocytes: Liver to endothelium – blood vessel,
• Highly efficient EC Gene Silencing: Kidney, liver, lung delivery of siRNA to multiple cell types. One Injection knocksout 5 genes
• endothelium – lunks, Hepatocytes
• Lung cancer targeted: effect the biology Kras and  of p53 – targeted RNA combination therapy
• Time altered tumor regression vs percent survival
• Cisplatin for survival comparison
• Immune cells for siRNA : CD45 vs control – different RNAs
• current siRNA Delivery Capabilities: lung, liver, leukocytes
• Transport aspects: nano-particles inside the cell: endocytosis – manipulate NPC-/- cells have increase siRNA retention, accumulation in late endosomes
• NPC: Other Modular Pharmacology –>>>>> mRNA vs siRNA
• mRNA – very big molecule – encapsulation: Formulation of mRNA transfection near 100% across multiple cell lines
• EPO – anemia to be fought – Production of EPO Protein via mRNA-LNP – collaboration with Shire
• increase of HCT by EPO
• Factor IX: himophilia
• CRISPR: Genetic repair inside cells: siRNA vs CRISPR: Cas9 Protein, guide RNA, DNA- repair template

11:30 – 12:00, 6/13/2014,  Daniel Anderson  “Intracellular RNA delivery”

@MIT – Summer Symposium 2014: RNA Biology, Cancer and Therapeutic Implications, June 13, 2014 8:30AM – 4:30PM, Kresge Auditorium @MIT

http://ki.mit.edu/news/symposium

REALTIME event coverage for the Scientific Media by Dr.  A. Lev-Ari 

in Open Access Scientific Journal of Leaders in Pharmaceutical Business Intelligence (LPBI)

http://pharmaceuticalintelligence.com

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Daniel Anderson, PhD 
Samuel A. Goldblith Professor of Applied Biology and Associate Professor, Chemical Engineering and Institute for Medical Engineering and Science, Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

Dr. Daniel G. Anderson is the Samuel A. Goldblith Professor of Applied Biology, Associate Professor, Chemical Engineering and Institute for Medical Engineering and Science, and member of the Koch Institute for Integrative Cancer Research at MIT. He received his PhD in molecular genetics from the University of California at Davis. At MIT, he pioneered the use of robotic methods for the development of smart biomaterials for drug delivery and medical devices. His work has led to the first methods rapid synthesis, formulation, analysis, and biological evaluation of large libraries of biomaterials for use in medical devices, cell therapy and drug delivery. In particular, the advanced drug delivery systems he has developed provide new methods for nanoparticulate drug delivery, non-viral gene therapy, siRNA delivery, and vaccines. His work has resulted in the publication of over 230 papers, patents and patent applications. These patents have led to a number of licenses to pharmaceutical, chemical and biotechnology companies, and a number of products that have been commercialized or are in clinical development.