Final Program - American Society of Gene & Cell Therapy

Program Schedule, Saturday, May 18, 2013
Scientific Symposium 434
1:45 pm - 3:45 pm
ROOM: 151 ABCG
Lessons Learned from the Manufacture and Clinical Translation of Gene and Cell Therapy
Products
CO-CHAIRS: Gene Liau, PhD and Gabor Veres, PhD
SPEAKERS
Barrie J. Carter, PhD
Overview on Product and Clinical Translation
After two decades of development, the irst gene therapy in the western world that was recently approved for marketing in Europe
was an rAAV, glybera. The progress over these two decades in developing and testing gene therapy products will be reviewed using
rAAV as the illustrative example. Substantial progress has been made in scaled up production of rAAV vectors, at least ive different
manufacturing systems have been employed to advance products into clinical testing. The puriication and quality testing of rAAV
vectors has advanced signiicantly and as more products approach licensing analytics for rAAV will be further reined. Pathways
for preclincal testing of rAAV for safety, toxicity and biodistribution are established. Importantly, clinical studies with rAAV have
generated clear evidence that clinical beneits can be gained and have also illuminated some important considerations such as host
immune responses.
Harold Petry, PhD
Lessons from the Glybera ® European Approval Process to Build a Gene Therapy Product Pipeline
The presentation will cover the development of Glybera ® , a gene therapy product based on the use of recombinant Adenoassociated
virus for gene delivery, designed for patients with Lipoprotein Lipase Deiciency. The European Medicines Agency’s
Committee for Medicinal Products for Human Use (CHMP) has approved authorisation of Glybera (alipogene tiparvovec) for
marketing in the European Union. It is intended to treat lipoprotein lipase (LPL) deiciency in patients with severe or multiple
pancreatitis attacks, despite dietary fat restrictions. Hurdles which were recognized during the development program towards
market authorization will be discussed.
Bruce L. Levine, PhD
Generation of T Cells that Engraft, Expand and Persist
Dr. Levine directs the Clinical Cell and Vaccine Production Facility and is an Associate Professor in the Department of Pathology
and Laboratory Medicine at the University of Pennsylvania. The CVPF develops, manufactures and tests novel cell and gene
therapies in clinical trials at Penn and collaborating institutions. Critical components for generation of cells with non-homologous
function include 1) source and isolation of cells, 2) method of activation, 3) method of expansion, 4) characterization and testing. T
lymphocytes can be activated, cultured, and endowed with novel functions ex vivo. Ex vivo modiied cells can engraft, signiicantly
expand in vivo by >1000 fold, mediate potent clinical responses, and can survive for years.
Raymond T. Bartus, PhD
CERE-120 (AAV-neurturin) for the Treatment of Parkinson’s Disease: Experience from 4 Clinical Trials and
Human Autopsy Data
CERE-120 is an AAV serotype 2 vector expressing the gene for human neurturin, a neurotrophic factor with the natural ability
to protect dying dopaminergic neurons in Parkinson’s patients from cell death and stimulate their ability to produce tyrosine
hydroxylase (TH), an enzyme necessary dopamine synthesis. Since CERE-120 can inhibit cell death and stimulate dopamine, it has
the potential ability to provide both symptomatic improvement as well as slow the neurodegenerative process, hence be disease
modifying. Ceregene has completed over 24 preclinical studies on CERE-120 in multiple animal models of Parkinson’s disease as
well as multiple preclinical toxicology studies. Ceregene has completed 4 CERE-120 clinical trials including 2 Phase 1 studies and 2
Phase 2/2b studies in Patients with moderately advanced Parkinson’s disease. Data from these studies as well as from the analysis
of 4 brains obtained at autopsy from subjects treat with CERE-120 will be discussed.
Saturday, May 18, 2013
Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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