Advances in gene therapy are driving uptake

Despite technical and regulatory hurdles, gene therapy is on its way to becoming a reality, according to a new report from business consultants Frost & Sullivan.

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 new study, "Advances in Gene Therapy", provides an overview of the current developments and advanced technologies in gene therapy.

"The positive results from cancer testing have positioned gene therapy closer to commercial roll-out," says Frost & Sullivan 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."

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, the report argues.

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," said Austin.