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View estimates of Gene Therapy Markets from 2005-2015 Today.


DUBLIN, Ireland -- Research and Markets (http://www.researchandmarkets.com/reports/c1595) has announced the addition of Jain PharmaBiotech's 'Gene Therapy - Technologies, Markets and Companies' to their offering

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA DNA: see nucleic acid.
DNA
 or deoxyribonucleic acid

One of two types of nucleic acid (the other is RNA); a complex organic compound found in all living cells and many viruses. It is the chemical substance of genes.
 are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo in vivo /in vi·vo/ (ve´vo) [L.] within the living body.

in vi·vo
adj.
Within a living organism.



in vivo adv.
 without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified genetically modified
Adjective

(of an organism) having DNA which has been altered for the purpose of improvement or correction of defects

genetically modified genetic adj [food etc] →
 cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense antisense, DNA or RNA manipulated in a laboratory so that its components (nucleotides) form a complementary copy of normal, or "sense," messenger RNA (mRNA; see nucleic acid).  techniques such as RNA interference RNA interference
n.
A process in which the introduction of double-stranded RNA into a cell inhibits the expression of genes.
 (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, non-viral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders This is a list of major and frequently observed neurological disorders (e.g. Alzheimer's disease), symptoms (e.g.back pain), signs (e.g. aphasia) and syndromes (e.g. Aicardi syndrome).  and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine veterinary medicine, diagnosis and treatment of diseases of animals. An early interest in animal diseases is found in ancient Greek writings on medicine. Veterinary medicine began to achieve the stature of a science with the organization of the first school in the , particularly for treating cats and dogs Cats and Dogs

A slang term referring to speculative stocks that have short or suspicious histories for sales, earnings, dividends, etc.

Notes:
In a bull market analysts will often mention that everything is going up, even the cats and dogs.
, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health of the US is playing an important part. Worldwide, there are 918 clinical trials of which two-third are in the US. In the US, there are protocols for 572 clinical trials excluding the gene marker studies and non-therapeutic trials, of which 200 are actively pursued currently. The largest number of clinical trial protocols (405 out of 572) is for cancer. The report identifies the areas for future research.

Since the death of Jesse Gelsinger Jesse Gelsinger (June 18 1981 - September 17 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. He was 18 years old. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver, whose  in the US following a gene therapy treatment, the FDA FDA
abbr.
Food and Drug Administration


FDA,
n.pr See Food and Drug Administration.

FDA,
n.pr the abbreviation for the Food and Drug Administration.
 has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency adenosine deaminase deficiency ADA deficiency A uniformly fatal AD disease, which consitutes 40% of Pts with SCID Clinical Cellular immune dysfunction, oral candidiasis, intractable diarrhea, FTT, severe diaper rash, pseudoachondrodysplasia, death by age 2 Lab . Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since January 2004. At least two products are expected to be approved by 2005 and gene therapy markets are estimated for the years 2005-2015. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.

Profiles of 183 companies involved in developing gene therapy are presented along with 209 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley John Wiley may refer to:
  • John Wiley & Sons, publishing company
  • John C. Wiley, American ambassador
  • John D. Wiley, Chancellor of the University of Wisconsin-Madison
  • John M. Wiley (1846–1912), U.S.
 & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies

Benefits of this report

- Up-to-date on-stop information on gene therapy with 67 tables and 12 figures

- Evaluation of gene therapy technologies

- 600 selected references from the literature

- Estimates of gene therapy markets from 2005-2015

- Profiles of 182 companies involved and collaborations in this area

Contents Include:

- Executive Summary

- Introduction

- Gene Therapy Technologies

- Clinical Applications of Gene Therapy

- Gene Therapy of Genetic Disorders The following is a list of genetic disorders and their origins. Beside most disorders is a code that indicates the type of fertilization and the chromosome involved.
  • P - Point mutation, or any insertion/deletion entirely inside one gene
 

- Gene Therapy of Cancer

- Gene Therapy of Neurological Disorders

- Gene Therapy of Cardiovascular Disorders

- Gene therapy of viral infections

- Research, Development and Future of Gene Therapy

- Regulatory, Safety and Ethical Issues of Gene Therapy

- Markets for Gene Therapy

- References

- Companies involved in Gene Therapy

Who should read this report?

- Biotechnology companies Top 100 Biotechnology Companies
The following is a list of the top 100 biotechnology companies ranked by revenue. The first nine companies qualify for the list of the top 50 pharmaceutical companies.
 developing gene therapy

- Academic institutions doing research in gene therapy

- Drug delivery companies

- Pharmaceutical companies interested in gene therapy

- Gene therapy companies

- Venture capital and investment companies

For more information visit http://www.researchandmarkets.com/reports/c1595
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Copyright 2005, Gale Group. All rights reserved. Gale Group is a Thomson Corporation Company.

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Publication:Business Wire
Date:Dec 14, 2005
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