A Special Report on Gene Therapy
A Special Report on Gene Therapy

Author: K. K. Jain

Publisher: John Wiley & Sons

Published: 2000-04-14

Total Pages: 308

ISBN-13: 9780471492900

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This special report on Gene Therapy Companies by K.K. Jain provides an up-to-date overview of the gene therapy industry. It contains a wealth of very practical information on 105 companies. For each company, information is available on the people to contact, their contact details, a corporate history, the company's technologies and products, financial data, patents held, products in clinical trials, collaborations with other companies and selected publications from the company's scientists. Almost 200 companies were initially considered for inclusion but just over 100 companies were finally selected as they fulfilled the necessary criteria to be classified as a 'Gene Therapy Company,', defined broadly to include cell therapy companies. Not all of the companies devote their activity exclusively to gene therapy but they have sufficient relevance to be included. As part of this project, new companies were discovered that have not been reported in other publications or on the internet. Dr. Jain has spared no effort to ensure that the information is up-to-date and accurate at the time of publishing. In most instances, the information was reviewed by the companies to correct any outdated information or to add any complementary or recent information. In addition to the detailed descriptions of each company, the data is also presented in summary tabular form so that at a glance the reader can identify collaborations within the gene therapy industry, or determine which companies are pursuing strategies involving viral, non-viral or other approaches to gene therapy, and which diseases are being targetted. Given that much of the gene therapy research and development is currently being carried out within the industrial sector, it is important for scientists, managers and investors to have the relevant information at their fingertips. This special report provides that information in one accurate, easy to consult and up-to-date source.

Exploring Novel Clinical Trial Designs for Gene-Based Therapies
Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2020-08-27

Total Pages: 127

ISBN-13: 0309672988

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Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

Oversight and Review of Clinical Gene Transfer Protocols
Oversight and Review of Clinical Gene Transfer Protocols

Author: Institute of Medicine

Publisher: National Academies Press

Published: 2014-03-27

Total Pages: 78

ISBN-13: 030929665X

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Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.

Human Genome Editing
Human Genome Editing

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2017-08-13

Total Pages: 329

ISBN-13: 0309452880

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Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.

Immunopharmacology
Immunopharmacology

Author: Manzoor M. Khan

Publisher: Springer Science & Business Media

Published: 2008-12-19

Total Pages: 275

ISBN-13: 0387779760

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During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.

From Research to Reality
From Research to Reality

Author: The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada

Publisher: Council of Canadian Academies

Published: 2020-11-05

Total Pages: 122

ISBN-13: 1926522796

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From Research to Reality describes the stages involved in the approval and use of gene therapies in Canada, and examines challenges associated with regulatory oversight, manufacturing, access, and affordability, and identifies promising approaches to address them.

Pharmaceutical Biotechnology
Pharmaceutical Biotechnology

Author: Daan J. A. Crommelin

Publisher: CRC Press

Published: 2002-11-14

Total Pages: 456

ISBN-13: 9780415285018

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The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.

Regulatory Aspects of Gene Therapy and Cell Therapy Products
Regulatory Aspects of Gene Therapy and Cell Therapy Products

Author: Maria Cristina Galli

Publisher: Springer

Published: 2015-09-15

Total Pages: 235

ISBN-13: 3319186183

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This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Novel Gene Therapy Approaches
Novel Gene Therapy Approaches

Author: Ming Wei

Publisher: BoD – Books on Demand

Published: 2013-02-13

Total Pages: 408

ISBN-13: 9535109669

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Gene therapy has the potential to be a tailor-made therapeutic with increased specificity and decreased side effects that can offer a "cure" for many disorders. The aim of this book is to provide up-to-date reviews of the rapidly growing field of gene therapy. Chapters cover a large range of topics including methods of gene delivery, and identification of targets with several papers on cancer gene therapy. If more people become aware of the true nature and potential of gene therapy, perhaps we can achieve the full benefit of such an innovative approach for the treatment of a range of diseases, including cancer.

Heritable Human Genome Editing
Heritable Human Genome Editing

Author: The Royal Society

Publisher: National Academies Press

Published: 2021-01-16

Total Pages: 239

ISBN-13: 0309671132

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Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight.