FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 63Program SchedulePresidential Symposium 330 — continuedJean Bennett, MD, PhDAn Aye for Gene Therapy: Lessons from a Clinical Trial for Congenital BlindnessGene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. The recent demonstrationof safe and stable recovery of retinal/visual function in 12 children and adults with Leber’s congenital amaurosis (LCA) due to RPE65 mutations in a trialbeing carried out at The Children’s Hospital of Philadelphia (CHOP), and encouraging results from two other clinical trials for LCA-RPE65 provide great hope for peoplewith other more common blinding diseases. This presentation will describe the latest safety and efficacy results in the LCA-RPE65 clinical trial being held at CHOP. Thisstudy is now >2.5 years past initiation. In addition, it will describe some of the challenges presented by the nature of the targeted disease itself, hurdles that havebeen navigated in order to conduct the study, and issues of importance for eventual approval of gene augmentation as a therapy for LCA and other blinding diseases.*This presentation involves contributions from the following additional individuals (listed alphabetically): Carmen Acerra, Defne Amado, Alberto Auricchio, SandroBanfi, Jeannette Bennicelli, Erin Bote, Yifeng Chen, Daniel Chung, Ann Fulton, Jeffrey Golden, Rebecca Grant, Bernd Hauck, Daniel Hui, Katherine A. High, BartLeRoy, Arkady Lyubarsky, Albert M. Maguire, Maureen G. Maguire, Kathleen Marshall, Federico Mingozzi, Jessica Morgan, Kristina Narfstrom, Stephen Orlin, EricA. Pierce, Settimio Rossi, Kenneth S. Shindler, Francesca Simonelli, Enrico M. Surace, Edwin Stone, Junwei Sun, Nasreen Syed, Francesco Testa, Nicholas J. Volpe,Jennifer Wellman, J. Fraser Wright, Gui-shuang Ying, Olga Zelenaia, Xiaosong (Sonia) ZhuFriday, May 21 stDavid A. Williams, MDNew Approaches to Gene Therapy for Fanconi AnemiaFA is a rare recessive syndrome characterized by bone marrow failure, congenital anomalies and a predisposition to malignancy. FA cells have a defect in DNA repairthat leads to increased spontaneous chromosomal breakage. Transgenic expression of FA genes in vitro corrects the increased chromosomal breakage and othercellular phenotypes of the disease. In a recent gene therapy trial, we have demonstrated that gene transfer per se is no longer a limitation to the effectiveness ofgene therapy in this disease. Rather our clinical studies show that even early in the disease process there are significant deficiencies in the number and/or functionof hematopoietic stem cells (HSCs) that can be collected and utilized as targets in gene transfer. One approach that could address this deficiency is the derivationof HSC from induced pluripotent stem (iPS) cells reprogrammed from non-blood somatic tissues. Important advantages of iPS cells for application of gene therapyare the apparent unlimited proliferative capacity of such cells (allowing production of large numbers of derived HSC) and the ability to clone such cells allowing bothsingle cell correction of genetic mutations and prospective molecular characterization of manipulated cells prior to therapeutic applications.
64American Society of Gene & Cell TherapyProgram ScheduleExhibit Hall Open & Networking Reception6:30 pm – 8:30 pmRoom: Exhibit Hall CFriday, May 21 stPoster Session II6:30 pm – 8:30 pmRoom: Exhibit Hall B South, CRNA Virus Vectors I(Abstracts 345 through 361; see pages 108-109)AAV Vectors II(Abstracts 362 through 377; see pages 109-110)Adenovirus and Other DNA Virus Vectors II(Abstracts 378 through 391; see page 110)Chemical and Molecular Conjugates II(Abstracts 392 through 407; see pages 110-111)Genetic and Metabolic Diseases Gene & Cell Therapy II(Abstracts 408 through 422 and Abstract 764 which has been moved from Poster Session III; see pages 111-112)Cardiovascular Gene & Cell Therapy(Abstracts 423 through 440; see pages 112-113)Ophthalmic Disorders and Technological Advances(Abstracts 441 through 453; see pages 113-114)Musculo-skeletal Gene & Cell Therapy II(Abstracts 454 through 468; see pages 114-115)Cancer – Immunotherapy II(Abstracts 469 through 487; see pages 115-116)Cancer-Oncolytic Viruses II(Abstracts 488 through 506; see pages 116-117)Cancer-Targeted Gene & Cell Therapy II(Abstracts 507 through 520; Abstract 519 withdrawn; see page 117)Hematologic and Immunologic Gene & Cell Therapy II(Abstracts 521 through 534; see page 118)Immunologic & Host Responses in Gene & Cell Therapy(Abstracts 535 through 554; Abstract 541 withdrawn; see pages 118-119)Oligonucleotide & RNAi Therapeutics II(Abstracts 555 through 569; see pages 119-120)Cell Processing and Vector Production II(Abstracts 570 through 579; see pages 120-121)Late Breaking Science II(Abstracts 933 through 937; see page 121)EXHIBITOR PROSPECTUSfinal program
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FINAL PROGRAM - American Society of Gene & Cell Therapy

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