GBI Research’s new report, “Gene Therapy Market to 2018- Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards”, provides in-depth analysis of the unmet needs, drivers and barriers that impact the global gene therapy market. The report analyzes technological aspects, advantages and disadvantages of various vectors used in gene therapy, and clinical development trends in gene therapy. It includes in depth analysis of the pipeline molecules, clinical development stages, promising pipeline molecules and therapy areas focused on in R&D. Furthermore, the report provides competitive benchmarking for the leading companies, and analyzes the mergers, acquisitions and licensing agreements that shape the global markets.
This report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by GBI Research’s team of industry experts.
Gene therapies are biological compounds, which modify or replace disease-causing genes. These therapies are the new therapeutic class aimed at treating diseases associated with genetic mutations. Gene therapy promises to provide new treatments for a large number of inherited and acquired diseases. The basic concept of gene therapy is to introduce a piece of genetic material into target cells, which will result in either a cure for the disease or a slowdown in the progression of the disease. It involves the transfer of a functional gene copy into specific cells of an individual in order to repair a faulty gene copy. It may be used to replace a defective gene, or to introduce a new gene to cure a condition.
For example, mutations in genes on the X chromosome lead to X chromosome-linked genetic diseases such as Duchenne muscular dystrophy and hemophilia. Since males have only one copy of the genes from this chromosome, there is no other normal copy available to fulfill a defective gene’s function which is present on the X chromosome. If the normal copy of the mutated gene is delivered in the nucleus externally through a delivery agent, the cells can produce the normal gene products and the disease would be treated.
From a commercial perspective, there is a huge unmet need in oncology and autoimmune diseases, amongst others, that could further drive growth of the pharmaceutical and biotech industry. The unmet need is largely driven by the lack of efficacious and safe therapeutic products based on conventional pharmaceutical and biotech research. Gene therapy is a new therapeutic category that has the potential to satisfy this unmet need, especially considering how efficacious and safe this therapeutic category is expected to be.
GBI Research’s analysis suggests that therapies developed using gene therapy technology can address the majority of the unmet needs prevailing in the current pharmaceutical market. The inherent structure of gene therapies and their potential to replace the functions of defective genes make them highly effective to knockdown any gene that was previously unapproachable by conventional therapies. Gene therapies are poised to become the next most promising class of drugs in the pharmaceutical industry. Currently there are only three approved products, namely Gendicine, Oncorine and Rexin-G, with a collective market little above $2.8m. Since first movers always have the competitive edge, many large pharmaceutical and biotechnology companies have already commenced their R&D activities on gene therapies.
This report provides insights into the major unmet needs prevailing in the current pharmaceutical industry, and points to gene therapies as the solution to these unmet needs. The report also elucidates the promising late-stage gene therapy pipeline, and provides insights into the gene therapeutics R&D pipeline and funding opportunities.
- Annualized various technologies and vectors used in gene therapy.
- Analysis of the leading therapeutic segments for which clinical development in gene therapy is being conducted.
- Key drivers and barriers that have a significant impact on the market.
- Coverage of pipeline molecules in various phases of drug development.
- Competitive landscape of leading companies. The key companies studied in this report are ReGenX Biosciences, Oxford BioMedica, EGEN, Inc, Sangamo BioSciences, BioSante Pharmaceuticals and Amsterdam Molecular Therapeutics.
- Key M&A activities and licensing agreements that have taken place between 2010 and 2011 in the global gene therapy market.