Satellite workshop6月14日（木）

Development of Human Embryonic Stem Cell Based Therapeutics for the Treatment of Degenerative DiseasesJane S LebkowskiActing Chief Scientific Officer: Regenerative Patch Technology

SW-1

Human Embryonic Stem Cells (hESCs) can proliferate indefinitely yet, upon appropriate cues, differentiate into all

somatic cell lineages. These two properties of hESCs enable the development of hESC-derived therapeutic cell

populations which can be batch manufactured in central manufacturing facilities, cryopreserved, and distributed

islet, osteoblast, chondrocyte, and hematopoietic cell populations which are functional in either in vitro or in vivo

animal models of human disease. For example, hESCs have been differentiated into oligodendrocyte progenitors that

upon transplantation into animals with spinal cord injuries, can remyelinate denuded axons, induce axonal sprouting,

and improve locomotor activity. Extensive preclinical studies have been completed to examine the activity,

biodistribution, dosing, delivery, and potential toxicity and tumorigenicity of the oligodendrocyte progenitors. The

safety of these cells is now being tested in the clinic in subjects with complete spinal cord injuries.

【略歴】［Current Position］Acting Chief Scientific Officer: Regenerative Patch Technology

［Education］

1977: BS Biology and Chemistry, Syracuse University 1982: Ph.D. Biochemistry, Princeton University 1985: Postdoctoral Fellowship Genetics, Stanford University

［Positions Held］

1986-1995: Vice President Research and Development, Applied Immune Sciences1995-1998: Vice President Discovery Research, Rhone Poulenc Rorer (currently Sanofi)1998-2012: Chief Scientific Officer and Sr Vice President Regenerative Medicine, Geron Corporation

トイラテサ

再生医療　日本再生医療学科雑誌　VOL.11 suppl 2012

SW-2

This talk will describe current FDA perspectives on scientific issues relevant to the use of cell and tissue-based

therapies, cover development of current FDA approaches to regulatory oversight in this area, and illustrate the roles

and responsibilities of the various FDA staff members in review of applications and formulation of policy.

The use of cell-based medical products promises to have a profound impact on public health. This talk will describe

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The regulatory and scientific approach used in the Office of Cellular, Tissue, and Gene Therapies and developed in

many parts of the FDA is designed to allow progress in clinical research while assuring patient safety and protection

of patient rights.

Satellite workshop6月14日（木）

Regulatory and Scientific Considerations for Cellular and Tissue-based Therapies

Steven BauerChief, Cellular and Tissue Therapy Branch Division of Cellular and Gene Therapies Office of Cellular, Tissue and Gene Therapies, CBER / FDA

【略歴】［Current Position］Chief, Cellular and Tissue Therapies Branch, Division of Cell and Gene Therapies, Office of Cellular, Tissue, and Gene Therapies, Center for Biologics Evaluation and Research, Food and Drug Administration (2005 -)

［Education］

1977 University of Maryland, College Park, BS Zoology1986 University of Maryland, College Park, PhD Biochemistry

［Positions Held］

1986 Scientific Member, Basel Institute of Immunology, Basel Switzerland1991 Senior Staff Fellow, CBER/FDA1998 Principle Investigator, Division of Cellular and Gene Therapies, CBER/FDA 2002 Chief, Laboratory of Stem Cell Biology, Division of Cellular and Gene Therapies, CBER/FDA2005 - Chief, Cellular and Tissue Therapies Branch, Division of Cell and Gene Therapies, CBER/FDA

トイラテサ

再生医療　日本再生医療学科雑誌　VOL.11 suppl 2012

Satellite workshop6月14日（木）

Quality and nonclinical aspects of Cell-based Medicinal Products - from a European regulator s perspective

Matthias RennerPaul-Ehrlich-Institut

SW-3

Rapid scientific progress in the fields of genetics, cell biology and biotechnology resulted in the development of novel

innovative cell-based therapies. In the European Union they belong to the class of Advanced Therapy Medicinal

Products (ATMPs), which are subject to a centralized authorisation procedure. A consolidated regulatory framework

for ATMPs has recently been established (Regulation 1394/2007 and Commission Directive 2009/120) and specific

Guidelines addressing scientific and technical requirements for the development of such medicinal products exist.

The European and national regulatory framework for cell-based medicinal products will be presented and specific

aspects and challenges in the quality and non-clinical development and assessment of these therapies will be

discussed.

【略歴】［Current Position］Scientific assessor and researcher, Department of Medical Biotechnology, Paul-Ehrlich-Institut, Langen, Germany (2009)Co-Chair, CAT Gene Therapy Working Party (GTWP), European Medicines Agency (EMA) (2010)Senior lecturer, University of Veterinary Medicine, Vienna, Austria (2007)

［Education］2008 Habilitation (Virology) University of Veterinary Medicine, Vienna, Austria 1991 to 1995 Ph.D. Max-Planck-Institute of Biochemistry, Department of Virus Research, Martinsried, Germanyy

［Positions Held］

1999 to 2008 Group leader, Institute of Virology, University of Veterinary Medicine Vienna,1997 to 2001 Group leader at Bavarian Nordic A/S, Vienna, Austria1995 to 1997 PostDoc, Max-Planck-Institute of Biochemistry, Department of Virus Research, Martinsried, Germany

トイラテサ

再生医療　日本再生医療学科雑誌　VOL.11 suppl 2012