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Biotech's next buzz: Gene therapy?

The next buzz in biotech
The next buzz in biotech

Imagine if instead of using drugs or surgery, a doctor could simply insert a gene into a patient's cell to treat or prevent disease.

Gene therapy is endeavoring to do just that and could emerge in the coming years as a viable alternative for treatment. Certain conditions that previously couldn't be treated, such as forms of blindness, types of cancer or more rare conditions like sickle cell disease are now being researched with this alternative treatment.

Phil Nadeau, biotechnology analyst with Cowen and Company, predicts there will be several gene therapy products approved by the F.D.A. within the next few years. He told CNBC recently he sees more than $1 billion in gene therapy sales worldwide, which ultimately means significant investments in gene therapy in the near future.

"In the past, most major companies stayed away from having gene therapy programs. In the future, we think, it's going to be a standard treatment," he said.

Safety challenges have historically been the largest barrier for gene therapy, Nadeau explained. For example, insuring that the insertion of new genes doesn't cause cancer or prompt other reactions have been on researcher's minds, but he's confident that more studies are easing concerns.

Spark Therapeutics, BioMarin and Avalanche Biotechnologies are companies currently in the space that have promising treatments coming out, Nadeau said.

"Spark Therapeutics is likely to have the first gene therapy program approved in the U.S. It's a recent IPO, but we think it's a really promising company."