"The first commercial gene therapy to provide an outright cure for a deadly disease."

Sounds like rare diseases are easier to cure

#Health

Sun, May 8th, 2016 11:00 by capnasty NEWS

The MIT Technology Review brings to attention a new treatment model that, using gene therapy, corrects flawed DNA in a patient, providing an "outright cure for a deadly disease." While the cure awaits approval, the real dilemma that pharmaceutical companies are facing is "how to bill for a high-tech drug that people take only once."

Kili says the general idea is to leapfrog from ultra-rare diseases to less rare ones, like beta-thalassemia, hemophilia, and sickle cell disease. However, he doubts the technology will be used to treat common conditions such as arthritis or heart disease anytime soon. Those conditions are complex, and aren’t caused by a defect in just one gene.

“Honestly, as we stand at the moment, I don’t think gene therapy will address all the ills or ailments of humanity. We can address [single-gene] disease,” he says. “We are building a hammer that is not that big.”

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