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San Jose, Calif.--May 2, 2003--As the dismal status of the entire biotech market dissolves numerous potentially lucrative partnerships, many companies developing gene therapy are focusing their R&D efforts on only a few key products.
The idea is to have enough therapies in the pipeline to balance inevitable failures, but not so many that they drain the already strained financial resources.
"The primary difficulty in developing clinically useful gene therapies has been designing a delivery system that delivers sufficient quantities of therapeutic DNA into enough cells to be expressed at levels that affect the disease being treated," states Technical Insights Analyst Katherine Austin.
Other approaches work by stimulating an immune response, mediating specific cell killing, activating a pro-drug, or producing a molecular decoy required for the replication of a virus. DNA vaccines are also in clinical trials for various forms of cancer and infectious disease such as herpes, hepatitis, and AIDS.
The techniques currently vying for the position of successful gene therapy delivery system include viral vectors, ex vivo cell transfection, liposomal vectors, artificial chromosomes, matrix vectors, genetically engineered cells, gene activators, bacterial vectors, and naked DNA.
Research is on to investigate methods such as transcription factors, antisense inhibition or RNA interference, and gene repair to regulate expression without the transfer of genes. Vectors that target particular tissue and cell types to avoid the systemic exposure and side effects such as those seen in current chemotherapy protocols are also in the pipeline.
As vector systems demonstrate success in clinical trials and clinical applications for gene therapy are developed, the cancer, vascular diseases and cystic fibrosis markets are likely to be the first to reap financial benefits from gene therapy.
"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 Austin.
New analysis by Technical Insights, a business unit of Frost & Sullivan (www.Technical-Insights.frost.com), Emerging Developments in Gene Therapy and DNA Vaccines, shows that the unstoppable innovative talent of the gene therapy community is ironing out the technical challenges facing this market. The study provides an overview of several companies involved in gene therapy R&D, details barriers to commercialization, and provides forecasts for disease sectors that are likely to benefit from this technology.
Technical Insights will hold a conference call at 1:00 p.m. (EDT)/ 10:00 a.m. (PDT) on May 8, 2003 to provide a summary and analysis of the latest developments in gene therapy and DNA vaccines. Those interested in participating in the call should send an email to Julia Paulson at jpaulson@frost.com with the following information for registration:
Full name, Company Name, Title, Contact Tel Number, Contact Fax Number, Email. Upon receipt of the above information, a confirmation/pass code for the live briefing will be emailed to you.
Frost & Sullivan is a global leader in strategic growth consulting. Acquired by Frost & Sullivan, Technical Insights is an international technology analysis business that produces a variety of technical news alerts, newsletters, and reports. This ongoing growth opportunity analysis of gene therapy and DNA vaccines is covered in Genetic Technology News Alert, a Technical Insights subscription service, and in Proteomics, a Frost & Sullivan Technical Insights technology report. Technical Insights and Frost & Sullivan also offer custom growth consulting to a variety of national and international companies. Executive summaries and interviews are available to the press.
Emerging Developments in Gene Therapy and DNA Vaccines
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