Final Program - American Society of Gene & Cell Therapy

Program Schedule, Wednesday, May 15, 2013
SPEAKERS
Anja Ehrhardt, PhD
Viral Hybrid-Vectors for Stable Tranduction of Mammalian Cells: Molecular Design, Eficacy and Applications
Viral hybrid-vectors combine the high transduction eficiencies of recombinant viruses with improved genetic elements for
stabilized transgene expression. This tutorial provides a state-of-the-art overview of available viral hybrid-vector systems for
random somatic integration (Sleeping Beauty transposase), targeted integration (designer nucleases) and episomal persistence
of the transgene (plasmid replicons). Advantages but also limitations of each vector system will be discussed and safety issues
addressed. Furthermore, recent in vivo applications will be mentioned.
Alberto Auricchio, MD
AAV - From the Genome to the Capsid
Vectors based on the adeno-associated virus (AAV) are being tested in humans and the initial safety and eficacy results in several
tissues, including liver, muscle, retina and central nervous system are extremely encouraging. The availability of hundreds of
naturally-occurring AAV serotypes, as well as of mutagenized variants with improved and different transduction proiles provide
a theoretical endless source of vectors for in vivo gene therapy. Sophisticated strategies were developed to tailor transgene
expression to speciic cell types within the AAV cargo capacity. In addition, efforts are being made to expand AAV cargo capacity
for the expression of large transgenes. Improvements on both the outside (capsid) and inside (genome) of AAV vectors will be
discussed in the context of the retina which is an excellent target tissue to challenge the versatile AAV platform.
Wednesday, May 15, 2013
Philip Ng, PhD
Helper-dependent Adenovirus: Design, Production and Applications
This education session will discuss optimal design, eficient production and novel applications of helper dependent adenovirus for
gene and cell therapy.
Lunch Break (On Own - Not Provided; Pre-ordered boxed lunches available for pick up at Registration Counter)
12:00 pm - 1:15 pm
Education Session 120
1:15 pm - 2:45 pm
ROOM: BALLROOM B
Emerging Field Review: Therapeutic Antibody Gene Delivery
Recent advances in monoclonal antibody discovery, isolation, and expression have created unprecedented opportunities to utilize this class
of proteins for broad therapeutic beneit. A number of studies have highlighted several potential applications for the use of antibodies
in vivo, ranging from the prevention of infectious diseases, to the treatment of drug addiction. This session will focus on several of these
applications and will highlight strategies aimed at maximizing vector-based expression of antibodies in vivo.
CO-CHAIRS: Alejandro B. Balazs, PhD and James M. Wilson, MD, PhD
SPEAKERS
James M. Wilson, MD, PhD
Inluenza Antibodies and Airway Delivery
Ronald G. Crystal, MD
Gene Transfer Strategies to Treat Addiction
Addiction to drugs is a major worldwide problem for which there is no effective therapy. Using cocaine and nicotine as examples
of commonly used addictive drugs, we have developed two platform strategies to evoke high-titer antibodies speciic for the antiaddictive
drug. In the irst strategy, based on the highly immunogenic nature of the adenovirus capsid, analogs of the addictive
drug are bound to adenovirus capsid proteins to create active vaccines that generate high-titer anti-drug antibodies. In the second
strategy, the gene coding sequences for monoclonals against the addictive drug are expressed by adeno-associated virus gene
transefer vectors capable of generating, with a single administration, persistent high titers of anti-drug antibodies. In experimental
animal models, both strategies are highly effective, providing new paradigms for anti-addictive molecular vaccines.
Alejandro B. Balazs, PhD
Expressing Antibodies in vivo via Adeno Associated Virus Vector for Vectored ImmunoProphylaxis
Recent advances in vector-based antibody delivery have created the potential for engineering humoral immunity to address
problems ranging from infectious disease to drug addiction. These efforts are often hampered by dificulties in achieving
therapeutic levels of antibody expression. This presentation will focus on means of improving expression of antibodies in vivo and
summarize the challenges of working with this novel application of gene transfer.
Final Program SALT LAKE CITY, UTAH May 15–18, 2013
39