Despite technical and regulatory hurdles, gene therapy is on its way to becoming a reality. Research and clinical trials have expanded to include cancer, neurological, cardiovascular, and pulmonary disorders, infectious diseases such as AIDS, and monogenic disorders such as cystic fibrosis and hemophilia. Short-term therapy markets such as cancer and cardiovascular disease will probably be the first to reap financial benefits from gene therapy.
Frost & Sullivan’s (ti.frost.com) new study, Advances in Gene Therapy, provides an overview of the current developments and advanced technologies in gene therapy, and a complete analysis of the key market drivers, restraints, applications, and trends that impact this field.
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“The positive results from cancer testing have positioned gene therapy closer to commercial roll-out,” says Frost & Sullivan Research Analyst Katherine Austin. “Moreover, the first gene therapy products, already in the late-stage clinical trials, are expected to rely on a short-term effect: for example, the killing of cancer cells through immune stimulation or induction of apoptosis (cellular suicide); or the growth of new blood vessels for the treatment of cardiovascular disease.”
Novel innovations in polymer chemistry, nanotechnology, and viral vectors, and an increasing understanding of the biology of exogenous gene expression, expect to overcome the technical obstacles and boost the growth of this technique. In addition, significant advances in recombinant DNA technology and genetic engineering have improved ways to introduce new genes into cells, resulting in the expansion of disease targets for gene therapy beyond traditional genetic diseases to chronic diseases such as diabetes.
However, a number of technical and regulatory hurdles are still prevalent, particularly for long-term gene therapies. To date, only a single human gene-therapy product, Gendicine, for the treatment of cancer in China, has reached the market. The main difficulty in developing clinically useful gene therapies has been in designing safe and efficient delivery systems. Regulatory barriers represent another major hurdle.
“The numerous requirements of the ideal vector include, the need to deliver sufficient quantities of therapeutic DNA into a large number of cells, to express the gene at sufficiently high levels to have an effect on the disease, and to do all this without toxicity,” explains Austin. “In addition, regulatory agencies in countries other than China are being extremely cautious, following a number of high profile cases where the gene therapy caused illness or death.”
There is still little doubt that scientific ingenuity will eventually overcome the remaining obstacles. Gene therapy now is about where monoclonal antibodies were fifteen years ago, and that market is currently worth over $20 billion a year. If developers can persevere and overcome the technical hurdles, major pharmaceutical companies will collaborate, investors will return, and once a few products prove to be safe, regulatory agencies will ease up.
Advances in Gene Therapy is part of the Technical Insights Subscription, and it thoroughly examines gene-delivery vehicles and technologies, vector design, DNA vaccine design, and clinical status of product candidates. This study also discusses the following markets: cancer, cardiovascular disease, genetic disease, infectious disease, neurological disorders, and other chronic and acute disorders. Interviews are available to the press.
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