Transfection and Gene Transfer: Technologies and Markets

Sequencing of the 80,000 - 100,000 genes in the human genome is near completion. Gene therapy is one of the many applications of these newly sequenced genes. For at least the last ten years in the gene therapy field, scientists have been focused on trying to transfer a gene to a cell, have the cell uptake the gene, and then have the gene produce a protein. Researchers, however, have not yet discovered the technology that will allow them to climb the gene transfer step. Directly harvested cells, cultured cell lines, genetically modified cell lines, viral vectors, and liposomes are all methods researchers use for ex vivo gene therapy experiments and in vivo gene therapy clinical trials. Viral vectors and liposomes are two types of technologies that are being developed which are allowing academic, government, and industry researchers to find ways to successfully transfer a gene for gene therapy.

Various estimates and forecasts of the global gene therapy market range from $1 billion to $5 billion in the next five years. The gene therapy market is projected to be $12 billion as early as 2007. A projection of $45 billion dollars for the gene therapy market in 2010 indicates continued rapid growth. All of the gene therapy forecasts are dependent, however, on finding successful gene transfer methods and continued government and consumer support of gene therapy research. To date, gene therapy is still a developing field. Gene therapy is not yet a revenue generating product. At this time, more than thirty companies and twenty research centers are focused on developing viral vector and liposome gene transfer technology for gene therapy and DNA vaccine applications.

In this study focusing on viral vector and liposome gene therapy gene transfer methods, Kalorama will investigate the various viral vector and liposome, gene transfer methods, potential markets, the companies and research centers developing viral vector and liposome gene transfer methods, and any role government and/or public policy has on the development of viral vector and liposome gene transfer technology.

For this discussion, the gene therapy transfer technology field can be divided into five key segments:

• Viral vectors
• Chemical vectors
• Physical methods
• Artificial/synthetic chromosomes
• Stem cells

This report focuses on the human gene transfer technology that is being developed, the options that the technologies present, and the progress being made in these fields. Gene transfer technology might have a major impact in the next five years, but long-term market effects will be established more gradually. The technology trends present an informative picture of advances in gene transfer technology.

Press Release

Nonviral Gene Transfer Technology to Make Gains on Viral Vectors

New York, January 22, 2002 /PR Newswire —Over the next few years, nonviral gene transfer technologies will make significant gains on viral vector methods in the race to the gene therapy marketplace, according to a new study released today by Kalorama Information and available at MarketResearch.com. Although virus-based technologies are still the most frequently used methods for effecting therapeutic gene transfer, nonviral methods are becoming more prevalent, and the two are predicted to reach near parity by 2005, according to the study.

The study, Transfection and Gene Transfer: Technologies and Markets, reveals that no single technology has yet emerged as the definitive gene transfer method. Indeed, quite the opposite is the case: according to the report, the field is showing increasing diversification, according to the report. And revenues for all types of methods are predicted to grow at a steady pace.

"In academic circles, viral technology still predominates," notes Laura Ruth, PhD, author of the report. "However, nonviral methods are experiencing more growth within industry, probably because the non-viral approaches are likely to be more practical in the near term." Non-viral methods include lipid, polymer, artificial chromosome, stem cell, and physical means.

Gene transfer technology development should have obvious market pay-off. More than 30 different acquired, monogenic, multigenic, short-term, and chronic diseases, each with potential values in excess of a billion dollars, are currently under study, and licensing revenues are steadily increasing, according to the study.

"Predictions of a gene-therapy revolution have proved to be premature in the past," cautions Steven Heffner, an acquisition editor at Kalorama Information. "Non-viral and viral phase III clinical trials in the cancer field look promising, but there is no guarantee that they will show efficacy. And since no one technology seems like a magic bullet, vector development across the board is heating up."