FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 41Program ScheduleThursday, May 20George Stamatoyannopoulos Lecture 2008:00 am - 9:15 amRoom: Marriott Ballroom Salons 1, 2 & 3George Stamatoyannopoulos LectureChairBarrie J. Carter, PhDSpeakerThursday, May 20 thHal E. Broxmeyer, PhDUnderstanding and Enhancing Stem/Progenitor Cell Function and EngraftmentThere are a number of cytokines, cytokine receptors, cell surface proteins, and intracellular molecules that regulate hematopoietic stem (HSC) and progenitorcell (HPC) function. Amongst these are the chemokine, stromal cell derived factor-1 (SDF-1/CXCL12), its receptor, CXCR4, the cell surface protein CD26/Dipeptidylpeptidase IV, which truncates and inactivates specific cytokines, the Rapamycin sensitive m-TOR pathway, and the sirtuin family member, SIRT1. Effortsare underway to modulate these proteins/molecules for enhanced engraftment of HSC/HPC and accelerated hematopoiesis.Exhibit Hall Coffee Social9:15 am – 10:30 amRoom: Exhibit Hall C
42American Society of Gene & Cell TherapyProgram ScheduleThursday, May 20 thScientific Symposium 21010:30 am - 12:30 pmRoom: Marriott Ballroom Salons 2 & 3Clinical TrialsChairR. Jude Samulski, PhDSpeakersSteven A. Rosenberg, MD, PhDCancer Immunotherapy Using Genetically Modified T CellsPatients with advanced cancer have been treated by the adoptive transfer of autologous T cells transduced with retroviral vectors encoding antitumor T cell receptors.Objective cancer regressions have been seen in patients with metastatic melanoma receiving cells targeting melanoma/melanocyte differentiation antigens and in patientswith synovial cell sarcoma who received cells targeting the NY-ESO-1 cancer-testes antigen. Clinical trials have also begun targeting CD19 on lymphomas and targetingcarcinoembryonic antigen on colorectal cancers.Ian MacLachlan, PhDA Phase I Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Lipid Nanoparticle ApoB siRNA (ApoB-SNALP) in Hypercholesterolemic SubjectsStabilized nucleic acid lipid particles (SNALP) are a modular lipid nanoparticle delivery platform developed for the delivery of nucleic acid based drugs. Two SNALP productcandidates, containing siRNA payloads, are currently being evaluated in clinical trials in oncology and metabolic indications. By the end of 2010 we expect that as many asfive SNALP based product candidates will be in clinical trials. SNALP containing siRNA that potently silence the conventionally non-druggable target, apolipoprotein B, havebeen evaluated in a Phase 1 clinical trial in hypercholesterolemic subjects. Early results, including the elucidation of previously cryptic immune stimulatory activity associatedwith the chemically modified siRNA payload, will be presented.Bill Hauswirth, PhDLeber Congenital Amaurosis (LCA) Gene Therapy Clinical TrialResults and implications of a recently initiated AAV vector clinical trial for LCA2 (recessive RPE65 mutations) will be discussed. LCA2 patients received 150ul (5.9 exp10vector genomes) of GMP grade AAV2-CBA-hRPE65 vector subretinally and their visual function then followed periodically over the next 18 months. No adverse eventswere noted for any patient, all demonstrated substantial and significant improvement in light sensitivity, up to 63,000 times better that pretreatment baselines, and somedeveloped a new preferred locus of visual fixation centered on the area of treated retina.R. Jude Samulski, PhDDesigning AAV Vectors for Translational Studies: Update on DMD Phase 1 TrialInitial AAV vectors for clinical studies have been derived from natural isolates. We have engineered and tested in Phase 1 DMD study chimeric vector derived from type 1and type 2. Outcome of these studies and next generation of DMD viral vectors suited for efficient gene delivery will be described and pre-clinical data presented.Scientific Symposium 21110:30 am - 12:30 pmRoom: Marriott Ballroom Salon 1Tissue Specific Stem CellsChairCarolyn Lutzko, PhDSpeakersPamela Itkin-Ansari, PhDDevelopment of Cell Based Therapies for DiabetesThe presentation will address a range of issues regarding development of pancreas specific stem cell based therapies. First, evidence for stem cells in the adult pancreas andsuggested methods for improved identification of such populations will be discussed. Strategies for the recruitment and differentiation of tissue specific stem cell populationsin vitro and in vivo will also be addressed. Finally, because insulin producing β-cells are cell autonomous (in contrast to many cell targets, e.g. neurons and cardiomyocytes),they are amenable to novel transplantation and regeneration approaches which will be presented.EXHIBITOR PROSPECTUSfinal program
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