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TUESDAY, NOVEMBER 3

12:30 pm Registration

Challenges in RNAi Delivery

2:00 Chairperson’s Opening Remarks

Christina Rondinone, Ph.D., Director, Research, Metabolic Diseases, Hoffmann-La Roche Inc.

2:10 Delivery is in the Details

Catherine J. Pachuk, Senior Director, Delivery Biology, Pfizer Research Technology Center

2:40 Stem Cells as a Non-Immunogenic Vehicle for Delivery of siRNA

Peter R. Brink, Ph.D., Professor and Chair, Department of Physiology and Biophysics, Stony Brook University

Although siRNA has the potential to be a unique therapeutic agent, delivery has been hampered by immune responses for siRNAs in the vascular or interstitial spaces. One potential delivery system are human mesenchymal stem cells (hMSCs) because they are not immunogenic and it has been shown that hMSCs can be injected into an organ (the heart) where they integrate into the tissue and form gap junctions with the target cells. Gap junctions are also capable of mediating transfer of siRNA from one cell to another. These features make for a viable cellular delivery system.                

3:10 Nanoparticles for Controlled and Targeted Delivery of siRNA

Mark Saltzman, Ph.D., Chair, Department of Biomedical Engineering, Yale University

3:40 Networking Refreshment Break in the Exhibit Hall

4:20 A Biodegradable, Biocompatible, Sustained Release System for RNAi Delivery

Seth D. Feuerstein, M.D., J.D., President, Carigent Therapeutics

While cationic and lipid delivery vehicles allow for encapsulation of high levels of RNAi material such as siRNA and miRNA, they are generally limited by cytotoxicity for direct applications as well as preference for delivery to the liver. Recently, polymer vehicles have begun to show promise for delivery of RNAi and can have specific and clear advantages such as delivering to different organs and less (or no) cytotoxicity. With high loading in non-cationic polymers that are FDA approved sustained release delivery can be achieved for direct applications such as, skin or systemically in organs such as the brain and lungs.

4:50 Highly Efficient Delivery of RNAi to Neurons in Vivo Using NeuroVex

Anna-Maria Anesti, Ph.D., Product Development Director, NeuroVex, BioVex Inc.

Suzanne Thomas, Ph.D., Director of Project Management, NeuroVex, BioVex Inc.

At BioVex we have developed a highly efficient method for gene silencing in neurons in vivo using NeuroVex disabled HSV-1 vectors. NeuroVex vectors allow delivery of shRNAs, artificial miRNAs, which are the new generation RNAi triggers with improved safety profiles for gene silencing in the nervous system, and multiple miRNAs in tandem for simultaneous silencing of multiple genes. Here, we show that NeuroVex delivery of shRNA/miRNA to sensory neurons in vivo results in highly effective and specific silencing of targeted genes. This has not been feasible using other vector systems and allows new approaches to in vivo gene target validation.

5:05 Conception of Circular Interfering RNA and Production’s Strategy
Guillaume Plane, CEO, MitoProd SA
Interfering RNAs represent a powerful tool for the Pharmaceutical Industry. Indeed, the stability, quality, in vivo efficiency, and delivery of these molecules represent major challenges for their development. Basically, by circularizing the primary transcript and thus removing extremities, MitoProd brings an innovative solution to face most of these challenges. In association with MitoProd innovative technology for RNA manufacturing, based on the fermentation of Saccharomyces cerevisiae yeast, Circular Interfering RNAs (ciRNA®s) offer some major advantages for the development of siRNA-based drugs.



5:20 Cell Type-specific siRNA Delivery and the Treatment of Disease
Derek M. Dykxhoorn, Ph.D., Assistant Professor, University of Miami School of Medicine
The delivery of siRNAs to the appropriate cell types and tissues in vivo remains one of the biggest hurdles being faced for the development of RNAi-based therapeutic approaches. Cell type-specific approaches have been developed which facilitate the targeted delivery of siRNAs to specific cell populations by taking advantage of the affinity of various molecules (antibodies, aptamers, etc) for cell surface antigens. These approaches have taken the promise of RNAi-based therapeutics one step closer to reality.  

5:50 Combinatorial Development of Synthetic siRNA Delivery Systems

Daniel Anderson, Ph.D., Research Associate, Massachusetts Institute of Technology

High throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on high throughput methods for developing and characterizing siRNA delivery systems. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated and screened for their ability to delivery siRNA, both in vitro and in vivo. A number of siRNA delivery formulations have been developed with in vivo efficacy, and show potential therapeutic application for the treatment of genetic disease, viral infection, and cancer.               

6:20 Close of Day

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