Abstract
Benefits of this report
- Up-to-date on-stop information on gene therapy with 72 tables and 13
figures
- Evaluation of gene therapy technologies
- 700 selected references from the literature
- Estimates of gene therapy markets from 2008-2018
- Profiles of 192 companies involved and collaborations in this area
Who should read this report?
- Biotechnology 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
Summary
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 are now being
introduced without the use of vectors and various techniques are being used to
modify the function of genes in vivo without gene transfer. If one adds to
this the cell therapy particularly with use of genetically modified cells, the
scope of gene therapy becomes much broader. Gene therapy can now combined with
antisense techniques such as RNA interference (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,
nonviral 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 and viral infections with
emphasis on AIDS. Applications of gene therapy in veterinary medicine,
particularly for treating cats and dogs, are included.
Research and development is in progress in both the academic and the
industrial sectors. The National Institutes of Health (NIH) of the US is
playing an important part. As of 30 July 2007, over 1340 gene therapy clinical
trials have been completed, are ongoing or have been approved in 28 countries.
The total number of human gene transfer trials in the US that are registered
with NIH Office of Biotechnology Activities is 885 as of December 2007. A
breakdown of these trials is shown according to the areas of application. The
largest number of clinical trial protocols is for cancer. The report also
identifies the areas for future research.
Since the death of Jesse Gelsinger in the US following a gene therapy
treatment, the FDA 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. 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 2008 and gene therapy
markets are estimated for the years 2008-2018. 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.The markets for DNA vaccines
are calculated separately as only genetically modified vaccines and those
using viral vectors are included in the gene therapy markets
The voluminous literature on gene therapy was reviewed and selected 670
references are appended in the bibliography.The references are constantly
updated. The text is supplemented with 72 tables and 13 figures.
Profiles of 192 companies involved in developing gene therapy are presented
along with 213 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 & 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.
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