Final Program - American Society of Gene & Cell Therapy

Program Schedule, Thursday, May 16, 2013
Scientific Symposium 212
10:30 am - 12:30 pm
ROOM: BALLROOM D
Frontiers of Cardiovascular Gene and Cell Therapy
CO-CHAIRS: David A. Dichek, MD and Michael J. Passineau, PhD
SPEAKERS
Charles R. Bridges, MD, ScD
Challenges of Gene and Cell Therapy to the Heart
In this presentation I will describe the main challenges of current cardiac cell and gene therapy with an emphasis on challenges to
eficient delivery. I will give a detailed comparison of available gene and cell delivery techniques. I will discuss documented and
potential complications of cell injections and gene transfer such as the risk of arrhythmias, incidence of coronary restenosis and
malignancy. Finally, I will focus on the most recent results of cardiac cell and gene therapy trials and the major lessons drawn from
these trials.
Thursday, May 16, 2013
Andrew H. Baker, PhD
Vascular MiRNA and Pulmonary Hypertension
MicroRNAs (miRNAs) are short non-coding RNAs which post-transcriptionally regulate gene expression. miRNAs are important
in governing cell differentiation, development and disease. We have investigated the role or individual miRNAs in pathological
vascular remodeling, as well as the potential therapeutic value of manipulating selected miRNA in vivo. I will discuss these
indings with emphasis on vascular remodeling associated with both venous and arterial remodeling, i.e. vein graft failure and the
development of pulmonary arterial hypertension.
Marc Penn, MD, PhD
Gene Therapy for Heart Failure
The success of establishing antegrade low in patients with acute myocardial infarction has led to signiicant advancements in the
survival of patients with acute ischemic events of the myocardium. Unfortunately this success had led to a signiicant increase in
the prevalence of heart failure. To address this need several investigators have turned to gene therapy as a strategy to either repair,
restore or optimize cardiac function. Excitingly these strategies have entered clinical trials with encouraging results. We will discuss
the strategies currently under investigation as well as those that maybe entering the clinical realm in the future.
Doris A. Taylor, PhD
Personalizing Cell Therapy: Initial CCTN Experience
Scientific Symposium 213
10:30 am - 12:30 pm
ROOM: BALLROOM B
New Paradigms for Immune Cell Therapies
CO-CHAIRS: Cynthia Dunbar, MD and Paris Margaritis, DPhil
SPEAKERS
David L. Porter, MD
Chimeric Antigen Receptor Therapies for Lymphoid Malignancies
This presentation will describe the biological activity and outcomes of patients treated with CART19 (CTL019) cells; these are
autologous T cells collected by leukapheresis and transduced with a lentivirus encoding anti-CD19 scFv linked to 4-1BB and CD3-z
signaling domains. Gene-modiied T cells can then target CD19+ cells. Cells are expanded and activated ex-vivo by exposure to anti-
CD3/CD28 beads prior to infusion. Patients are treated for advanced and/or refractory CLL or ALL. We previously reported marked
in-vivo expansion, long-term persistence and effective anti-tumor activity of CART19 cells in 3 patients with CLL with relatively
short follow up (Porter, et al NEJM 2011; Kalos et al Sci Trans Med 2011). Results from the clinical trials will be updated with longterm
follow-up and include presentation of data from a larger cohort of patients. We show that these cells can undergo massive
in-vivo expansion, persist for more than 2 years and remain biologically active, and can induce potent and sustained responses for
patients with advanced, refractory and high risk CLL and relapsed, refractory ALL.
Maria-Grazia Roncarolo, MD, PhD
Cell Therapy with Regulatory T Cells to Dampen Undesired Immune Responses
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Final Program SALT LAKE CITY, UTAH May 15–18, 2013