Abstract
Growing Pressure for Results Renews Investor Interest in Gene Therapy
Biotech companies in the United States are facing enormous pressure to develop a gene therapy approved for marketing. While China has achieved this milestone with the worlds first approved gene therapy, Gendicine, some participants have expressed doubts whether the initial studies conformed to U.S. standards in terms of patient numbers and duration of treatment. Nevertheless, there is a great sense of urgency among these companies to have an approved gene therapy, not only to bring hope to patients suffering from unique diseases, but also to present a strong case to potential investors. Introgen Therapeutics has come closest to achieving this. With many promising late-stage pipeline products, it is helping drive the gene therapy market and boost the credibility of the technology. Other companies with vector technologies are also approaching commercial success, and their growing visibility is expected to increase the confidence of companies with gene therapy targets.
This Frost & Sullivan research service provides a comprehensive analysis of the U.S. gene therapies market, examining the present performance of pipeline products at discovery and clinical development levels and evaluating future market potential. It tracks and discusses the most critical technological breakthroughs in gene therapy research. The study also examines vectors and delivery systems in the pipeline and analyzes them by type and application.
Competitor Activity Alerts Participants on Creating Intellectual Property Portfolio
With most biotech companies still years away from having an approved and marketed gene therapy, the need to generate value within existing technologies is critical. Building an intellectual property (IP) portfolio is one of the best ways of achieving this, since it creates value and a potential revenue stream as well as protects products and technological platforms. Companies could realize short-term revenues by licensing out the technology for other uses -- for instance, in the basic research sector.
"Not only is competition in the gene therapy market high, the industry is also extremely segmented," notes the analyst of this research service. "It is important that companies obtain substantive IP protection to increase their chances of survival and competitive advantage." Since results in the form of landmark achievements are reported to investors -- even if the products are still undergoing research and trials -- companies will have a strong opportunity to amass a significant amount of proprietary IP.
Expanding Knowledge Bases Leads to Sophistication in Manufacturing and Trial Protocols
Researchers are yet to create the ideal vector despite tremendous progress made in this area. Existing vector types are need-based and their use is determined by the target disease, the tissue, and the condition of the patient. For example, in a case where long-term transgene expression is required, a retroviral vector that integrates into the host cell genome is likely to be used. If long-term expression is not required, a vector that does not fuse with the target cell but exists as an episome (plasmid) within it is likely to be the appropriate choice.
"At present, there is a vast body of research on vectors that is constantly expanding," says the analyst. "This will undoubtedly lead to better designed vectors and improvements in manufacturing and incorporation into clinical trials." It is also expected to boost industry confidence in selecting and manipulating vector systems, as indicated by companies growing expertise in matching viral vectors with selected disease targets
View Cart
