Final Program - American Society of Gene & Cell Therapy

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Program Schedule, Friday, May 17, 2013 Robert M. Frederickson, PhD Molecular Therapy and the ASGCT Publishing Program This presentation will provide a brief overview of the Society’s lagship journal, Molecular Therapy, as well as the new online-only, open-access sibling journals. It will also present a roadmap of the editorial processes at the journals along with suggestions on how to navigate these processes successfully. Finally, we will outline opportunities for new investigators to get involved and contribute to the ASGCT publishing program. New Investigator Mentoring Event 8:30 pm - 9:30 pm ROOM: BALLROOM D Foundation Symposium 340 7:30 pm - 9:30 pm ROOM: BALLROOM A Cell and Gene Therapy in the Eye, Navigating the Road from the Lab to the Clinic Supported by the Foundation Fighting Blindness. CHAIR: Stephen M. Rose, PhD SPEAKERS Stephen M. Rose, PhD The Role of the Foundation Fighting Blindness in Bringing Treatments to the Clinic - Funding the “The Valley of Death” Sue Washer, MBA Small Company Experience; Maximizing Sources of Funding and Shots on Goal Samuel C. Wadsworth, PhD Large Company Experience, Navigating Through “New Technology” Challenge in a Large Organization New technological advancements drive development of new therapeutic modalities. Newness implies risk of failure which is the norm for scientiic endeavors because out of failure often comes insight that propels ields to new heights of understanding. Failure is an accepted ingredient of product development as well – obviously well-managed and early failure is ideal in a corporate environment. Layering on of new technologies such as gene and cell therapies complicates the process. Navigation through this arena will be discussed. Michael Young, PhD Retinal Stem Cells: Working with Biotech I will present my perspective on collaboration with biotech in the development of a stem cell therapy for retinal degeneration. My group has worked with Reneuron, Inc. for almost 8 years now, and we hope to translate our work into a clinical product . I will discuss the challenges one encounters when working closely with industry. Friday, May 17, 2013 Final Program SALT LAKE CITY, UTAH May 15–18, 2013 67
Program Schedule, Friday, May 17, 2013 Workshop 350 7:30 pm - 9:30 pm ROOM: BALLROOM C International Consortium for Gene and Cell Therapy Workshop CHAIR: Jacques P. Tremblay, PhD SPEAKERS Jacques P. Tremblay, PhD Introduction to the Creation of an International Consortium of Gene Dr. Tremblay has recently published in collaboration with 50 experts in gene therapy a letter in Molecular Therapy proposing the creation of an International Consortium of Gene Therapy to accelerate the development of clinical applications. Indeed the recent progress in genomics has permitted to identify the genes responsible for 10,000 hereditary monogenic diseases. Although each one of these diseases is rare (they are thus designated as orphan diseases), this whole group of diseases affects 8% of the human population and is thus a major cost for the public health systems. On the other hand, gene therapy has recently permitted successful treatment of some of these hereditary diseases. In particular, the new AAV serotypes permit to deliver a corrective gene more eficiently because human are not pre-immunized to some of the new serotypes. There are also several new tools to correct a mutated gene: meganucleases, ZFNs, TALENs and the CRISPR system, or intervene at the level of the mRNA: oligonucleotides for exon skipping, siRNA and shRNA. Promising results have been recently obtained in several animal models of hereditary diseases. The limiting step to be able to push these discoveries to a clinical application is the high cost of producing the clinical grade vectors. The workshop during the annual meeting of the ASGCT is to set the basis of that consortium, deine its goals and elect a direction committee. Among the proposed objectives will be to lobby governments for increase funding of this type of research, establishing international patient registry, developing more GMP facilities to produce the therapeutic agents, making the regulations more uniform in various countries. A large part of the workshop will be devoted to an open discussion so that ALL the participants can make their suggestions about the objectives and the methods to reach them. Yu-Quan Wei, MD, PhD Gene Therapy for Genetic Diseases in China Xiao Xiao, PhD Current Status of Clinical Grade AAV Vector Production Friday, May 17, 2013 Annemieke Aartsma-Rus, PhD The Treat-nmd Paradigm The TREAT-NMD Alliance is a network for the neuromuscular ield that provides an infrastructure to facilitate and accelerate the clinical development of new therapies. During its EU funded period (2007-2011) the network has collaborated with the USA to develop tools such as disease registries, standard of care guidelines, a clinical trial site registry, standardized operating procedures for preclinical animal studies, has set up the TREAT-NMD advisory committee on therapeutics (TACT), has maintained a biobank with samples from neuromuscular disorder patients and has gathered a wealth of information for the neuromuscular ield (see www.treat-nmd.eu). Following the end of the EU funding, the Alliance and its tools have been maintained and the network is running with other sources of funding. Current focus is on furthering the biobanks, setting up patient registries for additional neuromuscular disorders and in more countries, optimizing the clinical trial site registry, optimizing outcome measures for ambulant and non-ambulant patients and generating standards of care for additional diseases and updating existing care guidelines. The TREAT-NMD Alliance has also given rise to new EU funded projects such as RD-Connect, Neuromics, Optimistic and SKIP-NMD. While for the TREAT-NMD infrastructure the disease area is the connecting factor, it can serve as a paradigm for the gene therapy consortium where the therapeutic tool is the connecting factor. Philip J. Brooks, PhD Activities of the Ofice of Rare Diseases Research, NCATS, NIH, in Facilitating Translational Research in Rare Diseases Around the World 68 Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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ASGCT Officers and Board of Directo
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Annual Meeting Exhibitors - as of A
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Committee Rosters Advisory Council
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Committee Rosters Program Committee
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Abstract Reviewers Cancer - Immunot
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Outstanding New Investigator Awards
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Abstract Author Index Aartsma-Rus,
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Abstract Author Index Bartel, Ronnd
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Abstract Author Index Ugai, Hideyo.
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Final Program - American Society of Gene & Cell Therapy

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