Final Program - American Society of Gene & Cell Therapy

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Program Schedule, Wednesday, May 15, 2013 Workshop 140 7:30 pm - 9:30 pm Room: 150 DEFG Commercialization Workshop CO-CHAIRS: Estuardo Aguilar-Cordova and Brendan Lee, MD, PhD SPEAKERS Peter Kolchinsky What makes Investors want to Invest Nick Leschly Who Utilizes Early Phase Resources Wednesday, May 15, 2013 Fabrizia Bignami, PhD What is Big Pharma Looking for in Gene or Cell Therapy Suzannah Sundby, JD What IP Issues are Relevant for Successfully Commercializing Gene and Cell Therapies Gene and cell therapies are different from classic small molecules in that intellectual property issues can evolve and become increasingly complex as scaling up from the laboratory setting and commercialization proceeds. What effect do modiications and optimizations to vector designs and manufacturing processes, including cell isolation and culturing techniques, have on one’s right to make, use, and sell the intellectual property Do you still own the rights to the intellectual property Will the intellectual property rights of another prevent you from commercializing your own product Ms. Sundby will discuss IP in the cell and gene therapy space with particular attention paid to what questions an investigator or institution should consider prior to investing further resources in pipeline development. New Member Welcome Reception 7:30 pm - 8:30 pm ROOM: 255 B Final Program SALT LAKE CITY, UTAH May 15–18, 2013 45
Program Schedule, Thursday, May 16, 2013 George Stamatoyannopoulos Lecture 200 8:30 am - 9:35 am ROOM: BALLROOM E-J George Stamatoyannopoulos Lecture CHAIR SPEAKER Thursday, May 16, 2013 David W. Russell, MD, PhD Mario R. Capecchi, PhD Modeling of Human Disease in the Mouse in the 21st Century: From Cancer to Neuropsychiatric Disorders Gene targeting allows the designed modiication of any gene in the mouse genome. Since genes impact all biological phenomena this methodology can be used to study any biological phenomena common to mammals in the mouse. We are using it to model human disease in the mouse. The models can be used to analyze the pathology of the disease at a level not feasible in humans and as a platform for the development of new therapeutic protocols. I will discuss modeling of a neuropsychiatric disorder, obsessive compulsive (OCD) spectrum disorder in the mouse. These studies provide the unexpected conclusion that microglia, the immune cells of the brain derived from bone marrow, are controlling behavior in the mouse. Exhibit Hall Coffee Social 9:35 am - 10:30 am ROOM: EXHIBIT HALL C/D Scientific Symposium 210 10:30 am - 12:30 pm ROOM: 151 ABCG 50 Years of Research in Alpha-1 Antitrypsin CHAIR: Terence R. Flotte, MD SPEAKERS Robert A. Sandhaus, MD, PhD Historical Review of the Past 50 Years of Research on Alpha-1 Antitrypsin Disorder First described 50 years ago, Alpha-1 Antitrypsin Deiciency (AATD) was soon associated with the development of both lung and liver diseases. The rapidity with which the biochemical, pathophysiologic, epidemiologic, and therapeutic advances followed this initial discovery has been astounding, even by today’s standards. In spite of this, there is still much to be learned about the alpha-1 antitrypsin protein and its roles in health and disease. Ronald G. Crystal, MD Gene Therapy for Alpha 1-antitrypsin Deiciency with AAV Intrapleural Administration a1-antitrypsin (a1AT) deiciency, caused by SERPINA1 gene mutations, results in low a1AT serum levels and development of earlyonset panacinar emphysema, reducing life span by 15 to 20 years. Current treatment is weekly intravenous administration of a1AT puriied from pooled human plasma. Adeno-associated (AAV)-mediated delivery of a1AT is an ideal strategy for gene therapy for the lung manifestations of a1AT deiciency because AAV vectors mediate sustained transgene expression. We have established a novel, highly eficient strategy for gene therapy for a1AT deiciency based on intrapleural administration of AAV serotype rh.10 coding for human a1AT. The advantage this approach offers is proximity of vector expression to the site of action. Pre-clinical studies comparing intrapleural administration of 25 AAV serotype vectors demonstrated that the highest a1AT levels were achieved with AAVrh.10, a serotype that is derived from a non-human primate, minimizing the challenge of pre-existing human anti-vector immunity limiting persistent gene expression. Safety studies in mice and non-human primates have shown this strategy is safe and will provide sustained expression a1AT. Our overall hypothesis is that a single intrapleural administration of an AAVrh.10 gene transfer vector coding for the human a1AT cDNA will provide sustained therapeutic levels in the lung suficient to treat a1AT deiciency. 46 Final Program SALT LAKE CITY, UTAH May 15–18, 2013
Page 2 and 3: ASGCT Officers and Board of Directo
Page 4 and 5: Annual Meeting Exhibitors - as of A
Page 6 and 7: Annual Meeting Supporters Patron Le
Page 8 and 9: Committee Rosters Advisory Council
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Page 12 and 13: Committee Rosters Program Committee
Page 14 and 15: Abstract Reviewers Cancer - Immunot
Page 16 and 17: Outstanding New Investigator Awards
Page 18 and 19: Faculty List Thomas W. Chalberg, Jr
Page 20 and 21: Faculty List Ann M. Leen, PhD Baylo
Page 22 and 23: Faculty List Evan Y. Snyder, MD PhD
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Abstract Author Index Aartsma-Rus,
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Final Program - American Society of Gene & Cell Therapy

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