bluebird bio has received Breakthrough Therapy designation from the U.S. Food and Drug Administration for Lenti-D, its potential treatment for a rare and life-threatening neurological disorder in boys
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A man suffering from a rare genetic disease has been treated with a novel genome-editing therapy at the University of North Carolina’s Clinical and Translational Research Center.
Read MorePfizer plans to invest roughly $5 billion in capital projects in the U.S. over the next five years, including upgrading and expanding U.S. medicine factories and adding the capability to make gene therapies.
Read MoreThe treatment is part of an emerging field of medicine that could produce dozens of new gene-targeting medications in the next few years.
Read MoreUniversity of North Carolina, a powerhouse in gene therapy research, has signed exclusive licensing agreements with a New York City biotechnology company for three preclinical gene therapies that could treat an eye disease, certain muscular dystrophies and rejection of corneal transplants.
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