FINAL PROGRAM - American Society of Gene & Cell Therapy

58American Society of Gene & Cell TherapyProgram ScheduleFriday, May 21 stScientific Symposium 31410:30 am - 12:30 pmRoom: Delaware SuiteNew Physical Gene Delivery System and VectorologyChairShulin Li, PhDSpeakersChristian Plank, PhDNanomagnetic Nucleic Acid DeliveryPhysical force has been used with great success to enable, enhance or localize nucleic acid delivery. In magnetofection, which is one such method, vectors for nucleic aciddelivery are associated with magnetic nanoparticles to accumulate or retain them at target sites with gradient magnetic fields. This concept of magnetic drug targetingenhances the efficiency of the delivery process over several orders of magnitude. This we have exploited to generate an integrated method of magnetic cell separation andgenetic modification, to boost the efficacy of oncolytic adenovirus and for establishing a tumor vaccine to treat feline fibrosarcoma. In other recent approaches we focus onextending the concept of magnetofection with thermo-sensitive magnetic liposomes, magnetic microbubbles and magnetic aerosols for magnetically guided drug delivery.Declan M. Soden, PhDEndoscopic Electroporation: Targeted Gene Delivery to Intraluminal TissuesThe minimally invasive control of primary tumours is a critically important part in the treatment process as it relieves symptoms, forestalls complications, facilitates responsivenessto systemic therapies and is often curative. We have developed a device, which allows for an endoscopic cancer treatment, specifically targeting colorectal, gastricand oesophageal cancers by applying a brief electric pulse to the tumour. This greatly enhances tissue permeability maximizing the local/tumor absorption of therapeuticagents (drugs or DNA). Absorption occurs only in the area that has been treated with the electrical field and therefore is targeted to the tumor, leaving surrounding healthytissues unaffected. Our research has examined the efficacy of the device for targeted gene delivery to intraluminal tissues in large animals. We have compared the transfectionefficacy of a standard CMV driven reporter gene (b-galactosidase) in both murine and porcine tissues. In addition we have examined the efficacy of a DNA vectorcoding for a viral RNA polymerase encoded from the Semliki Forest Virus in comparison to the CMV driven vector.The treatment of spontaneous canine colorectal cancers has also been conducted using the device to facilitate local drug (bleomycin) absorption after tumor tissueelectroporation. The results have demonstrated the treatment to be effective and safe with complete tumor ablation noted in the two inoperable cases treated to date.All procedures were conducted as a simple colonoscopy type procedure with no adverse side effects recorded. A phase I clinical study with the device is due to commenceearly 2010 to treat patients with inoperable rectal cancer and electrochemotherapy.Kate E. Broderick, PhDDermal Electroporation Devices for Prophylactic DNA Vaccine DeliveryDuring the presentation, Dr. Broderick will discuss novel prototype dermal devices developed by Inovio and the pre-clinical Immunogenicity data generated from thesedevices in a number of animal models.Laurence JN Cooper, MD, PhDNon-viral Gene Transfer into Human T CellsNon-viral approaches can be used to genetically modify T cells for therapy. We have combined electroporation with the Sleeping Beauty (SB) transposase/transposon DNAplasmid system to introduce desired transgenes. By improving the efficiency of electroporation with designer devices and improving the efficiency of integration by adaptingthe SB system for human translation, we seek to implement physical gene transfer systems for clinical trials.EXHIBITOR PROSPECTUSfinal program