Why Gene Therapy Is The Best Bet On Restoring Functional Vision

Shares of Spark Therapeutics Inc ONCE are up more than 35 percent since its IPO in late January. The company is engaged in the area of gene therapy and is developing products to restore eyesight.

Jeffrey Marrazzo, Spark Therapeutics CEO, was on CNBC Tuesday to shed more light on what the company does.

Potential To Restore Functional Vision

"The promise here is, by fixing the underlying blueprint of what a cell needs to function properly, you can actually restore, potentially restore [...] functional vision and do so potentially in a long-lasting effect," Marrazzo said.

"And what we have seen in patients [...] was that over many, many years we actually see the ability of that improved restoration of functional vision as well as retinal sensitivity actually maintaining itself."

Related Link: Gene Therapy Stocks Take Beating On Bad News for Celladon

Small Details, Big Differences

Marrazzo was asked about the recent controversy, concerning a similar product, that gene therapy doesn't provide permanent or long-lasting solutions. He replied, "Most importantly, one of the things that we have now been able to educate people about the last couple of weeks is that there are lots of small details that really add up to big differences in these products and gene therapy.

"So, things for example like the formulation of the way we put our product together. We showed in some preclinical studies that you can lose up to 80 percent of your product on a needle if you didn't actually formulate it in a certain way and we were doing that, whereas others were not. So, those details really matter," Marrazzo concluded.