Gene Therapy Technologies, Applications and Regulations

Gene Therapy Technologies, Applications and Regulations From Laboratory to Clinic Edited by Anthony Meager Division of Immunobiology, The National Institute for Biological Standards and Control, South Mimms, UK The development of gene-based technologies has been rapid over the past decade and has consequently resulted in a surge of interest in human gene therapy, the deliberate transfer of genes to somatic cells to cure or alleviate disease symptoms. Hundreds of clinical protocols involving variously designed vectors for efficient gene transfer have been developed. However, the use of such complex 'gene medicines' containing potentially heritable genes has raised numerous concerns regarding quality, efficacy and safety. Encompassing recent developments in the field and addressing current concerns this book:
* surveys many of the current technologies for preparing vectors for use in gene therapy protocols
* reviews the application of gene-mediated therapies to a range of medical conditions
* considers the regulatory aspects of gene therapy including product quality and safety requirements
* appraises the transfer of technologies from laboratory to clinic with regard to the attendant requirements and facilities for:
* good laboratory practice (GLP) conditions in the R&D laboratory
* large-scale production methods and good manufacturing practice (GMP)
* current in-process and final product testing

Written by international experts knowledgeable about many aspects of human somatic gene therapy, this book will be an essential guide for those embarking on gene therapy technologies relevant to specifications of production and testing of products (and procedures) required to meet existing regulations, including quality, efficacy and safety considerations.In der Biomedizin ist die Gentherapie zur Zeit das wohl aufregendste Forschungsgebiet. Sie verfügt über ein vielversprechendes Potential, große medizinische und therapeutische Fortschritte in den kommenden Jahren zu erzielen, wie z.B. die Behandlung von Krankheiten, die bislang als unheilbar galten oder sehr schwierig zu behandeln waren. In diesem maßgeblichen und umfassenden Band sind zum ersten Mal aktuelle Daten zusammengetragen worden, die unterteilt sind in Vorschriften von Gesundheits- und anderen Behörden, Gesetze und Primärliteratur. Enthalten sind Informationen über Technologien der Gentherapie sowie dazugehörende Bestimmungen über behördliche Auflagen von gentechnischen Produkten und Verfahren. Dieser Band leistet einen wichtigen Beitrag zu diesem aktuellen Thema, für das bislang keine maßgebliche Literatur zur Verfügung stand. (10/99)
Cationic Liposomes for Gene Therapy Applications (J. Clancy & E. Sorscher).

DNA Condensation and Receptor-mediated Gene Transfer (A. Ziady & T. Ferkol).

Retroviral Vectors (W. Günzburg & B. Salmons).

Lentiviral Vectors (A. Lever).

Adenoviral Vectors (S. Connelly).

Adeno-associated Viral Vectors (T. Flotte & B. Carter).

Advances in Engineering HSV Vectors for Gene Transfer to the Nervous System (M. Soares, et al.).

Mammalian Artificial Chromosomes: Prospects for Gene Therapy (T. Ebersole & C. Farr).

Gene Therapy for Severe Combined Immunodeficiency (A. Thrasher, et al.).

Gene Therapy for Haemophilia (R. Hoeben, et al.).

Cystic Fibrosis: Gene Therapy Approaches (N. Caplen).

Therapeutic Approaches to Haemoglobinopathies (Y. Beuzard).

Gene Therapy Approaches to Duchenne Muscular Dystrophy (S. Murphy & G. Dickson).

Lysosomal Storage Diseases (L. Lashford, et al.).

Prospects for Gene Therapy of HIV Infections and AIDS (C. Lee, et al.).

The Developemnt of the Regulatory Process in Europe for Biological Medicines: How it Affects Gene Therapy Products (A. Meager, et al.).

Developement and Regulation of Gene Therapy Drugs in Germany (K. Cichutek).

The Transfer of Technology from the Laboratory to the Clinic: In Process Controls and Final Product Testing (F. Borellini & J. Ostrove).

Good Laboratory Practice in the Research and Development Laboratory (A. Shepherd).

Facilities for Large-scale Production of Vectors Under GMP Conditions (J. Boyd).

Addendum.

Index.

"It succeeds in putting together, possibly for the first time, gene therapy vectors, clinical applications, and regulatory production issues." (Human Genetics, April 2000)

Anthony Meager is a Principal Scientist in the Division of Immunology and Endocrinology, The National Institute for Biological Standards and Control, UK. Following his PhD in Biochemistry, he has carried out research in biochemistry, cell biology, immunology, virology, and cytokines, especially interferons and tumor necrosis factor. He joined the NIBSC in 1980. Currently, he heads laboratories involved in the biological standardization of IFN and TNF, and research studies on the bioactivity and immunogenicity of cytokines. From 1990, he has also contributed to regulatory guidance on gene therapy, both for the European Medicines Agency and the World Health Organization.

Gene Therapy Technologies, Applications and Regulations From Laboratory to Clinic
Edited by Anthony Meager Division of Immunobiology, The National Institute for Biological Standards and Control, South Mimms, UK

The development of gene-based technologies has been rapid over the past decade and has consequently resulted in a surge of interest in human gene therapy, the deliberate transfer of genes to somatic cells to cure or alleviate disease symptoms. Hundreds of clinical protocols involving variously designed vectors for efficient gene transfer have been developed. However, the use of such complex 'gene medicines' containing potentially heritable genes has raised numerous concerns regarding quality, efficacy and safety. Encompassing recent developments in the field and addressing current concerns this book:

• surveys many of the current technologies for preparing vectors for use in gene therapy protocols
• reviews the application of gene-mediated therapies to a range of medical conditions
• considers the regulatory aspects of gene therapy including product quality and safety requirements
• appraises the transfer of technologies from laboratory to clinic with regard to the attendant requirements and facilities for:
• good laboratory practice (GLP) conditions in the R&D laboratory
• large-scale production methods and good manufacturing practice (GMP)
• current in-process and final product testing

Written by international experts knowledgeable about many aspects of human somatic gene therapy, this book will be an essential guide for those embarking on gene therapy technologies relevant to specifications of production and testing of products (and procedures) required to meet existing regulations, including quality, efficacy and safety considerations.