Report Overview
To Succeed, Gene Therapy Must Overcome Early Setbacks
Gene therapy has suffered some serious setbacks in the last few years and progress has been considerably slower than many people - particularly investors - had hoped for. The well-publicized death of one patient and the development of leukemia in three others have made it increasingly clear that careful, time-consuming research into the basic mechanisms of getting the DNA into cells, while avoiding immune attack, is essential. With more researchers joining the fray and many promising drugs in clinical trials expected to appear in the market by 2006, the potential applications for gene therapy are expanding.
This Technical Insights' study explores the various types of technologies under development for improved gene delivery. It gives an overview of several leading companies and other research institutions involved in gene therapy R&D, detailing barriers to commercialization and providing forecasts for disease sectors that are likely to benefit from this technology.
Research and Development Produces Promising Results
"Once the first successful products are on the market, numerous others are sure to follow, and investor enthusiasm will most likely equal that currently enjoyed by monoclonal antibody therapies," says Dr. Katherine Austin, the analyst for this study. Some of the disorders for which gene therapies are in development - in part because they are most amenable to treatment - include hemophilia, cystic fibrosis, cardiovascular disease, and cancer. Other approaches seek to stimulate an immune response, mediate specific cell killing, activate a pro-drug, or produce a molecular decoy required for the replication of a virus.
DNA vaccines are also in clinical trials and may eventually be developed for various forms of cancer and infectious diseases such as herpes, hepatitis, and AIDS. Technology is being developed to allow vectors to target particular tissue and cell types. The obvious advantages of this include the fact that systemic exposure and side effects as seen in current chemotherapy protocols can be significantly reduced.
Several Methods Strive to be the Lead Technology
"A number of techniques are currently the top contenders for the race for the perfect gene therapy delivery systems," says Austin. These include viral vectors, ex vivo cell transfection where patient cells are removed, genetically modified, and re-administered, liposomal vectors, artificial chromosomes, matrix vectors, genetically engineered cells, gene activators, bacterial vectors, and naked DNA.
In addition, there are alternate therapeutic approaches that regulate gene expression without the transfer of genes. These methods include transcription factors, antisense inhibition, or RNA interference, and gene repair. As vector systems demonstrate success in clinical trials and clinical application of gene therapy is developed, the markets for treating cancer, vascular diseases, and cystic fibrosis are expected to be the first to reap financial benefits from gene therapy. | 
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