ESGCT/ICH Workshop on Viral Vector Shedding ESGCT/ICH Workshop on Viral Vector Shedding • Introductory notes: What is vector shedding? Why is it important? Klaus Cichutek Chair, CHMP GTWP at EMEA Paul-Ehrlich-Institut, 63225 Langen, Germany E-mail: cickl@pei.de Rotterdam, 30 October 2007 CHMP GTWP CHMP GTWP Paul Paul- -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
• Dr. M. Papaluca-Amati and Dr. O. Maki-Ikola, European Medicines Agency • Prof. G. Dickson, ESGCT President, and the ESGCT Board • Prof. G. Wagemaker, Rotterdam, and the local Organizing Committee of the XVth Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) • Dr. Odile Cohen-Haguenauer, Coordinator, and Clinigen Network of Excellence, a European Network for the Advancement of Clinical Gene Transfer and Therapy CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
/EFTA ICH- - ICH Gene Therapy Therapy Gene Discussion Group Group Discussion CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
• Update from the ICH Regions • Issues • Harmonisation of technical requirements CHMP GTWP CHMP GTWP Paul Paul- -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
Dr Alex Kuta Dr Teruhide Yamaguchi Vice President Head Regulatory Affairs Division of Biological Chemistry and Biologicals Genzyme Corporation National Institute of Health Sciences 500 Kendall Street Tokyo Cambridge, MA Dr Teruyo Arato Dr Daniel Takefman Review Director Chief, Gene Therapy Branch Office of Biologics Office of Cellular, Tissue & Gene Therapies Pharmaceuticals and Medical Devices Agency Food and Drug Administration Tokyo Center for Biologics Evaluation and Research Dr Daisuke Maeda 1401 Rockville Pike Reviewer Rockville, MD Office of Biologics Dr Stephanie Simek Pharmaceuticals and Medical Devices Deputy Director Agency (PMDA) Office of Cellular, Tissue & Gene Therapies Tokyo 100-0013 Food and Drug Administration Mr. Takeo Ozawa Center for Biologics Evaluation and Research President & CEO Rockville, MD POC Clinical Research, Inc. Tokyo Dr Beth Hutchins Director Bioanalytical & Protein Chemistry Dept. Dr. Yoshiyuki Tanaka Schering Plough Biopharma Palo Alto Director Clinical Science Planning and Development Dr Anthony Ridgway Clinical Development Institute Sr. Regulatory Scientist Banyu Pharmaceutical Co., Ltd. Office of the Director Tokyo Center for Evaluation of Radiopharmaceuticals and Biotherapeutics Biologics and Genetic Therapies Directorate Ms. Maki Tanaka Health Canada Project Manager Ottawa, Ontario Research and Development Division M's Science Corporation Dr Andreas Marti Kobe Head Gene Therapy Clinical Trials & Products Division Biotechnology Medicines CHMP GTWP CHMP GTWP Swiss Agency for Therapeutic Products Paul- Paul -Ehrlich Ehrlich- -Institut Institut Swissmedic Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines Berne
• Dr. S. Simek, CBER-FDA, Prof. K. Cichutek and the ICH Gene Therapy Discussion Group (EU, US-FDA, MHLW/JPM, Health Canada, EFTA) Professor Dr Klaus Cichutek Vice President Paul-Ehrlich-Institute Langen Dr Outi Maki Ikola instead of Dr Marisa Papaluca Amati Scientific secretary of Gene Therapy Working Party (GTWP) Safety and Efficacy of Medicines European Medicines Agency – EMEA Canary Wharf, London E14 4HB Dr Sharon Longhurst Medicines and Healthcare products Regulatory Agency London Dr Richard Pilsudski Directeur Scientifique et Affaires Réglementaires AstraZeneca 1, Place Renault Rueil-Malmaison CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
What is Gene Therapy? What is Gene Therapy? viral or non-viral vector/ DNA therapeutic transgene/ vector (preventive) gene product in vivo diagnostic CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
What is Shedding? What is Shedding? therapeutic release of product from subject/patient (preventive) to whom the shedding product has been in vivo administered diagnostic product in gene therapy: - vector or - cell CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
What may be shed in gene therapy? What may be shed in gene therapy? therapeutic release of product from subject/patient to whom the product has been administered: preventive shedding - viral or non-viral vector particle/ in vivo micro-organism diagnostic - vector nucleic acid - genetically modified product in gene therapy: cell - vector - virus derived from/ or mobilising/ - cell recombined with vector CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
In- -vivo vivo gene gene transfer transfer In using vectors vectors using in vivo 1) local or systemic application 2) biodistribution CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
In- -vivo vivo gene gene transfer transfer In using vectors vectors using in vivo • biodistribution into bodily fluids including excreta • infectious or non-infectious agent • transmission to third parties: humans, animals 1) local or systemic application • recombination with other agents: 2) biodistribution viruses, micro-organisms, TSE agents • spread in humans CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
Which shed products/organisms pose a risk? Which shed products/organisms pose a risk? release of product from - infection of other subject/patient to whom human beings or the product has been animals: administered: - risk to individual: shedding adverse effects - viral /non-viral - risk to public: vector particle/m.-org. new pathogenic - vector nucleic acid organism - genetically modified cell - virus/micro-organism derived from/ mobilising/ recombined with vector CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
What may be the result of shedding? What may be the result of shedding? transmission to other human beings release of transmission to product from animals subject/patient to whom the shedding product has been administered transmission (from reservoirs) to micro-organisms, viruses, TSE-agents, plants CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
Definition of shedding shedding in in gene gene therapy therapy Definition of • Shedding in the field of gene therapy may be defined as • dissemination and/or transmission of • gene therapy product or product-related vector or infectious agent • from bodily fluids and excreta of the treated subject or patient • to humans other than the patient. CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
Considerations with with respect respect to to Considerations shedding in in gene gene therapy therapy shedding • What is shed? • How and whereto is something shed? • Does shedding lead to dissemination/transmission? • What are the consequences of dissemination/transmission? • How can the risk of shedding be assessed • in non-clinical studies, • in clinical studies • in post-marketing studies? • What kind of measures can be recommended to • assess shedding and/or • to reduce harmful consequences of shedding? CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
European Medicines European Society of Gene European Network of Agency and Cell Therapy (ESGCT) Excellence in Gene Therapy (EMEA) EMEA/ICH WORKSHOP ON VIRAL/VECTOR SHEDDING Tuesday 30 October 2007 (11.00-18.30) in conjunction with: The XVth Annual Congress of the European Society of Gene and Cell Therapy 27-30 October 2007 Rotterdam, The Netherlands CHMP GTWP CHMP GTWP Paul- Paul -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
ESGCT/ICH Workshop on Viral Vector Shedding ESGCT/ICH Workshop on Viral Vector Shedding • Introductory notes: What is vector shedding? Why is it important? Klaus Cichutek Chair, CHMP GTWP at EMEA Paul-Ehrlich-Institut, 63225 Langen, Germany E-mail: cickl@pei.de Rotterdam, 30 October 2007 CHMP GTWP CHMP GTWP Paul Paul- -Ehrlich Ehrlich- -Institut Institut Federal Agency for Sera and Vaccines Federal Agency for Sera and Vaccines
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