Final Program - American Society of Gene & Cell Therapy

Program Schedule, Friday, May 17, 2013
Judy Lieberman, MD, PhD
Aptamer-siRNA Chimeras Targeting CD4 Cells Inhibit HIV Transmission
One attractive strategy for interrupting HIV transmission is a topical microbicide, but the need for application around the time of
sexual intercourse leads to poor patient compliance. Intravaginal application of CD4 aptamer-siRNA chimeras (CD4-AsiCs) targeting
the HIV coreceptor CCR5, gag and vif protected humanized mice from sexual transmission. In nondividing cells and tissue, RNAimediated
gene knockdown lasts for several weeks, providing an opportunity for infrequent dosing not temporally linked to sexual
intercourse, when compliance is challenging. In follow-up studies, we investigated the durability of gene knockdown and protection,
protection afforded by CCR5 or HIV gene knockdown on their own, and effectiveness of CD4-AsiCs formulated in a gel in polarized
human cervicovaginal explants and in humanized mice. These data, showing persistent gene knockdown and sustained protection
that was improved in a gel formulation, suggest that CD4-AsiCs are a promising approach for developing an HIV microbicide.
Scientific Symposium 314
9:45 am - 11:45 am
ROOM: 151 ABCG
Realizing the Potential: Getting to Funding, Getting to Approval and Getting to Market
CO-CHAIRS: Anthony T. Cheung, PhD and Karen Kozarsky, PhD
SPEAKERS
Sander van Deventer, MD, PhD
Development of a Commercial Gene Therapy Product
The market authorization of Glybera by the EMA has given development of gene therapies a signiicant boost. In this presentation
I will discuss regulatory and inancial challenges associated with the development of (future) gene therapy products, which is
essential for successful transition of academic projects into commercial products.
David Kirn, MD
Oncolytic and Immunotherapeutic Vaccinia Viruses for Cancer: Clinical Proof-of-concept with JX-594
(Pexa-Vec)
This talk will be an overview of oncolytic and immunotherapeutic vaccinia viruses for cancer, with a focus on clinical proof-ofconcept
data with JX-594 (Pexa-Vec). Dr. Kirn will review this emerging novel therapeutic class, including the scientiic rationale
for cancer-selectivity, mechanisms-of-action, and human clinical trial data from Phase 1 and 2 studies with Pexa-Vec. Clinical trial
data will include pharmacokinetics, pharmacodynamics, tumor biopsy analyses, safety, anti-cancer eficacy and survival. Data from
a randomized dose-inding Phase 2 trial will be presented, demonstrating a dose-related overall survival beneit of high dose Pexa-
Vec treatment in patients with advanced liver cancer (Nature Medicine 2013).
Samuel C. Wadsworth, PhD
Realizing the Potential: Getting to Approval and Getting to Market; Focus on Orphan Indications
The successful path to approval and market for new drugs is a series of effective decisions. Basic science, clinical research and
manufacturing expertise represent some of the major stepping stones on this path, although there are many others. Academic
scientists, regulators, corporate scientists, regulators, patients and patient advocacy groups all play their roles on the journey. For
new technologies like cell and gene therapy, the beacon that should guide us throughout this journey should be the demonstration
of truly transformative beneit to patients.
Friday, May 17, 2013
Artur Isaev, MD, MBA
Development, Clinical Trials and Registration of Neovasculgen® Gene Therapeutic Drug
Presentation will cover the key steps in “Neovasculgen” VEGF165 plasmid gene therapy drug development and commercialization
starting from choosing a target disease to its marketing, including assessment of key characteristics of the future drug,
determination of the therapeutic approach to be used and design and performance of preclinical studies. The main focus of
the talk would be on “Neovasculgen” clinical trials design for chronic lower limb ischemia and their main results, which would
highlight the necessity of precise deinition of the end points to obtain objective data about safety and eficacy of the drug. Finally,
the presentation would summarize speciicity of drug development in Russia, current stage of “Neovasculgen” development, and
perspectives of plasmid gene therapy in general.
PANEL DISCUSSION
MODERATOR
Estuardo Aguilar-Cordova, PhD
Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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