FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 27Program ScheduleWednesday, May 19Scientific Symposium 1008:30 am - 11:30 amRoom: Maryland SuiteCell Therapy BioProcessing SymposiumCo-ChairsBoro Dropulic, PhD & Thomas TillettSpeakersDavid L. Urdal, PhDManufacturing of Sipuleucel-T for the Treatment of Men with Metastatic, Castrate Resistant Prostate CancerSipuleucel-T is an autologous active cellular immunotherapy for men with late stage prostate cancer. It is comprised of a patient’s peripheral blood mononuclear cells, includingantigen presenting cells that have been cultured with a recombinant antigen delivery cassette composed of prostatic acid phosphatase and granulocyte-macrophagecolony stimulating factor. The development of sipuleucel-T will be discussed and the manufacture of this novel product candidate will be described.Gary C. du Moulin, PhD, MPHIncorporating the Concepts of Modern Quality Systems into Cell-based Product DevelopmentWell understood manufacturing processes that identify critical sources of variability can lower risk and lead to safe and efficacious treatment modalities that consistentlydeliver the intended therapeutic performance. The evolution of pharmaceutical quality systems and its focus on patient safety brings the application of a scientific approachand quality risk assessment into practice. This presentation introduces developers to the principles of risk management and quality by design, concepts that can assist ininsuring that quality attributes are identified early in the product design phase and incorporated into the control of critical processes for cell and gene therapy products.Wednesday, May 19 thJane S. Lebkowski, PhDDevelopment of Human Embryonic Stem Cells for Therapeutic ApplicationsHESC-based regenerative cell therapies require 1) evidence for reliable production and quality control of product manufacturing, 2) rigorous safety testing in preclinicalmodels, and 3) the design of clinical trial protocols that assess the safety and benefit of the therapy in appropriate patient populations. GRNOPC1 is a population ofallogeneic cells containing oligodendrocyte progenitors derived from characterized, dedicated, human embryonic stem cell banks. GRNOPC1 induces myelination of axonsin rats with spinal cord injuries and in Shiverer mice, which lack compact myelin, and also produces numerous neurotrophic factors such as midkine, BDNF, and activin.Extensive preclinical studies were performed to determine the distribution of GRNOPC1 as well as any potential toxicities after injection near the thoracic injury epicenter.Based on this IND application, the FDA has granted clearance for Geron to initiate a human clinical trial to assess the safety of GRNOPC1 in patients with subacute,complete ASIA A, thoracic injuries whose last fully preserved neurological level is T3 to T10.Madhusudan V. Peshwa, PhDUse of Flow Electroporation to Load Human Cells to Deliver Clinically Relevant Genes for Cell TherapeuticsThe ability to specifically enhance/augment biological function of cells using safe, non-gene modifying technologies with ability to deliver the product at clinical/commercialscale using automated, regulatory-compliant, cost-effective processes overcomes the key hurdle in developing “effective” cellular therapies. This presentation providesan overview of our experiences in development and characterization of cellular products using immune cells and stem cells that have been engineered to deliver clinicallyrelevant potency/biological activity. Specific case studies discussed will include clinical/commercial delivery of a (1) a patient-specific cellular vaccine marketed as anOncology therapy in Japan and (2) an engineered endothelial stem cell product for treatment of pulmonary arterial hypertension (PAH) in phase I/II trials in Canada.Mitchell Finer, PhDChallenges in the Manufacturing Scale-Up of hES Cell Derived TherapeuticsHuman Embryonic Stem (hES) Cells have been traditionally derived and passaged as adherent clumps of cells on mouse or human fibroblast feeder layers. In order toproduce the quantities of cells sufficient for preclinical development and human clinical trials at an acceptable quality standard, methodology must be developed to enablexeno product free/serum free culture of hES cells as single cells in the absence of feeder lines. Further, while growth on plastic can support limited preclinical and clinicaldevelopment in some indications, achieving the scale of hES cell amplification necessary to treat diseases such as Type 1 Diabetes that require significant cell numbers(~100 billion differentiated cells), the ability to expand ES cells in suspension in the absence of differentiation, and the ability to synchronously differentiate large numbersof cells is required for product development in Type I Diabetes. Data describing the generation of feeder-free cGMP derived hES cell banks, expansion of large scalehES cell cultures in the undifferentiated state and scaled differentiation in suspension of hES-cells into pancreatic b-cell progenitors will be presented.
28American Society of Gene & Cell TherapyProgram ScheduleWednesday, May 19 thMichelle Williams, PhDDevelopment of Prochymal, an Adult MSC TherapyOsiris has been developing stem cell therapies to treat unaddressed medical needs since 1992. An important part of this process has been navigating the regulatorypathway, particularly through work in the lead indication, graft-versus-host-disease. In 2005, Osiris secured the first ever Fast Track and Orphan Drug designations fromFDA for a stem cell product, followed by Orphan drug designation in Europe as well. In 2008, FDA and Health Canada granted approval for a pediatric expanded accessprogram, providing all children with severe GvHD access to our stem cell drug, Prochymal. The late stage clinical development process is an instructive case study ofdevelopment of a novel therapy for a orphan indication.Scientific Symposium 1018:30 am - 11:30 amRoom: Marriott Ballroom Salon 1A Global Gene Therapy RenaissanceChairJohn Rasko, MBBS, PhDSpeakersDe-Chao Michael Yu, PhDClinical Development of KH901, a Conditionally Replicating Oncolytic Adenovirus Encoding Human GM-CSF, in Patients with Recurrent Head andNeck CancersKH901 is a conditionally replicating oncolytic adenovirus, in which the viral E1 genes are under the control of a modified telomerase promoter and a cDNA gene of granulocytemacrophage colony-stimulating factor (GM-CSF) is inserted in the E3 region. Preclinical studies have demonstrated that KH901 selectively replicates in telomeraseupregulatedand E2F-positive tumor cells and has strong anti-tumor activity. A total of 110 patients with recurrent head and neck cancer have been enrolled into clinicaltrials in which KH901 was given intratumorally while chemotherapy was the standard of care. KH901 was found to be well tolerated and safe while a significant immuneresponses and pro-longed progression-free survival were observed when compared to the chemotherapy group.Christoph Klein, MDGene Therapy for Wiskott-Aldrich SyndromeWiskott-Aldrich syndrome (WAS) is a primary immunodeficiency disorder associated with thrombocytopenia, eczema and autoimmunity. We treated ten WAS patients bytransfusion of autologous, genetically modified hematopoietic stem cells (HSC) between 2006 and 2009. We demonstrate sustained WAS protein (WASP) expressionin HSC, lymphoid and myeloid cells, and platelets after gene therapy. T and B lymphocytes, natural killer (NK) cells, and monocytes were functionally corrected. Thepatients´ clinical condition markedly improved with respect to hemorrhagic diathesis, eczema, autoimmunity, and predisposition to severe infections. Genome-wide insertionsite analysis demonstrated vector integration that targeted multiple genes controlling growth development and immunological responses in a persistently polyclonalhematopoiesis.Yasutomo Nasu, MD, PhDProstate Cancer Gene Therapy in Japan - from HSV-tk to REIC/Dkk-3Intraprostatic therapeutic gene transduction (in situ gene therapy) is one of the potent therapeutic option for prostate cancer gene therapy aiming at antimetastatic benefitsthrough the generation of immune cell-mediated cytotoxic activities that affect not only the primary tumor but also metastatic lesion. Many clinical trials with HerpesSimplex Virus Thymidine Kinase (HSV-tk) gene therapy have been conducted in many institutes showing clinical safety and efficacy including Japanese trial (MolecularTherapy 15:834, 2007). Also immunomodulatory in situ gene therapy using adenoviral Interleukin-12 gene is under way in Japan. As a preclinical study, new therapeuticgene REIC/Dkk3 has been proved to be potential therapeutic gene for prostate cancer as it has induces local apoptosis and systemic immune activation. (Cancer Research65:9617, 2005, Cancer Gene Therapy 14:765,2007, Int J Oncol 34:657,2009, J Bio Chem 2009 online). In this symposium ongoing development of in situ genetherapy for prostate cancer will be reported with regard to systemic immune activation.EXHIBITOR PROSPECTUSfinal program
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FINAL PROGRAM - American Society of Gene & Cell Therapy

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