Final Program - American Society of Gene & Cell Therapy

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
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Final Program SALT LAKE CITY, UTAH May 15–18, 2013