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Gene Therapy: Medicine and Commerce for the 21st Century


Description: Gene therapy is being actively researched in almost every serious disease.The 21st Century is upon us and the sequencing of the human genome has brought incredible challenges concerning how this information will be used. The number of discrete genes in the human being has been estimated to be between 100,000 and 140,000, most of which have already been isolated. Although the vast majority have not been functionally identified, numerous genes have been directly implicated in disease function or inherited disorders. For decades, scientists have dreamed of curing such catastrophic conditions in the most simple and elegant manner - by replacing these malfunctioning genes. That dream is now becoming a reality.Gene Therapy: Medicine and Commerce for the 21st Century is the sequel to the well-acclaimed second edition of the Human Gene Therapy Report. Focusing on the most recent developments in this entirely new world facing the pharmaceutical, medical and investment communities, this report is aimed at the business and science professional alike. This Gene Therapy Report will help the reader understand the science of gene therapy along with a realistic view of the field and its future. It will also show how independent companies and those affiliated with Big Pharma are pursuing paths to the prize that will, once and for all, demonstrate the ellusive proof of principal.Unique Benefits of this Report:1. Introduction to the functioning of genes and gene therapy, providing the background needed to put key issues into perspective2. Description of both disease-targeted preclinical and clinical research, giving insight into the novel trials in several disease areas3. Analysis and comparison of several gene delivery research methods, helping the reader decide which is most appropriate4. Examination of the critical areas of gene therapy research, giving the reader an understanding of advances yet to come5. Discussion of the ethical issues surrounding gene therapy research, educating the reader in this area of research and the clinical manifestations of its outcomes6. Exploration of the impact of current Uspto regulations on intellectual property and of stacking patents on the industry and its future7. Discussion of current investor attitudes and the business models now favored by gene therapy companies, giving the reader insight into where to proceed in this industry8. Comprehensive analysis of the regulatory ramifications of the industry, helping the reader avoid legal pitfalls and consequences9. Many gene therapy and business information resources are provided, helping the reader keep up-to-date in this fast paced, ever changing environment10. In-depth profiles of 34 business and scientific gene therapy companies, identifying chief positions and strategy insights


Contents: 1 Executive Summary

2 Introduction
Scientific Background
Historical Background
Summary of the Second Edition: Human Gene Therapy

3 Ethical Perspectives

4 Genetic Diseases and Disorders Linked to Identifiable Genes
The Use of Gene Therapy in Disease
Cancer
Cardiovascular Indications
Blood Disorders
Neurological Indications
Muscular Dystrophy
HIV
Pulmonary Disease
Bone and Tissue Disease
Autoimmune and Inflammatory Diseases
Lysosomal Storage Diseases
Mitochondrial Gene Therapy
A Listing of Clinical Trials in Other Monogenic Diseases
Clinical Trials Summary

5 Current Methodologies in Gene Therapy
Experimental Research
Delivery, Delivery, Delivery
The Regulation of Gene Expression

6 The Business of Gene Therapy
The Market
Big Pharma and Biotech
The View from Venture Capital
Wall Street
Intellectual Property Issues

7 NIH Research Programs
Intramural Research
Extramural Research

8 Regulatory Ramifications
FDA, NIH, and RAC
International Regulatory Activities

9 Related Technologies
Epigenetics
Antisense Therapy
Peptide Nucleic Acids
Stem Cell and Tissue Therapy
Transgenics

10 The Future of Gene Therapy

11 Gene Therapy Companies

12 Associated Companies and Suppliers

13 Appendices
Patents
NIH Research Grants
SBIR/STTR Awards
Organizations Supporting Gene Therapy
Gene Therapy Institutes
Gene Therapy and Related Journals
Other Internet Sites of Interest
Selected Bibliography




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Please fax this form to:
(646) 607-1907 or (646) 964-6609 - From USA
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