Final Program - American Society of Gene & Cell Therapy

Program Schedule, Wednesday, May 15, 2013
Coffee Break
7:30 am – 8:00 am
Scientific Symposium 100
8:00 am - 10:00 am
ROOM: BALLROOM D
Challenges and Success of Gene Therapy Product Approval
CO-CHAIRS: Dale G. Ando, MD and Barry J. Byrne, MD, PhD
SPEAKERS
Dale G. Ando, MD
Clinical Studies of the Infusion of ZFN CCR5 Modiied Autologous CD4 T cells (SB-728T) in HIV Subjects
CCR5 is the second receptor for HIV in R5 trophic HIV strains and the CCR5Δ32 gene mutation confers resistance to HIV infection in
humans. This resistance to HIV infection can be recreated in CD4 T cells by ex-vivo modiication of the CCR5 gene locus by speciic
Zinc Finger Nucleases Clinical studies treating HIV subjects with autologous CD4 T cells modiied at CCR5 by ZFN (SB-728T) in
order to protect against HIV have been performed. Clinical data on the pharmacology, immunologic effects and safety of these
treatments will be presented.
Wednesday, May 15, 2013
Theresa Chen, PhD
CBER Regulatory/Preclinical Considerations for AAV Products
The presentation will provide an overview of the current CBER perspective regarding the preclinical evaluation of AAV-based gene
therapy products and discuss regulatory and scientiic challenges in the safety and activity assessment of these investigational
products. According to 21 CFR 312.23 (a)(8), adequate information derived from pharmacology and toxicology studies is needed
to support a clinical investigation that is reasonably safe and scientiically feasible. Thus, the data from preclinical studies should
provide a better understanding of the activity, safety, vector biodistribution, and transgene expression proiles following product
administration in appropriate animal models. These data will then be used to guide aspects of the design of early phase clinical
trials of AAV-based products, including: 1) determination of a potentially safe starting dose level and dose escalation scheme; 2) an
optimal route of administration/anatomic site of delivery; 3) subject eligibility criteria; and 4) an adequate clinical monitoring plan.
Barry J. Byrne, MD, PhD
Next Generation Therapies with AAV
Evan Y. Snyder, MD PhD FAAP
The Rationale Use of Cell-based Therapies - Viewpoint from a Scientist who Advises Regulatory Agencies
Although it is true that regulatory agencies, when assessing applications for cell-based therapies, tend to focus primarily on issues
of safety, they are inevitably cognizant of the rationale behind the use of particular cell type in a particular clinical context in order
to determine whether the risk is justiied. In other words, they do pay attention to whether a presumed mechanism-of-action
is scientiically valid and supportable by the data. Certainly the scientists and physicians who advise such regulatory agencies
scrutinize such data. This talk will discuss both the rational and irrational use of cell therapy, particularly for the neurological
disorders, including a number of misconceptions.
Scientific Symposium 101
8:00 am - 10:00 am
ROOM: BALLROOM C
From Blood to the Brain: Gene and Cell Therapy of Genetic Diseases and Inborn Errors of
Metabolism
CO-CHAIRS: Randy J. Chandler, PhD, MB and Denise Sabatino, PhD
SPEAKERS
Ian E. Alexander, MBBS, PhD
Pre-Clinical Gene Therapy for Urea Cycle Disorders
A critically important function of the liver is the detoxiication of ammonia via the urea cycle. Infants with severe urea cycle
enzyme deiciencies commonly present early in life with life-threatening hyperammonemia, progressing to coma and death in
the absence of medical intervention. Effective management remains challenging and necessitates liver transplantation. Ornithine
transcarbamylase (OTC) deiciency, the most common urea cycle defect, has long been recognized as an ideal model for livertargeted
gene therapy. This presentation will explore contemporary efforts to treat OTC and other urea cycle defects, placing these
Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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