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An announcement from uniQure ( (QURE) ) is now available.
uniQure N.V. has announced the U.S. FDA’s Orphan Drug Designation for their investigational gene therapy AMT-191, targeting Fabry disease, a rare genetic disorder. This status can lead to benefits such as tax credits and seven years of market exclusivity upon approval. AMT-191, designed to produce the deficient enzyme in Fabry disease via a one-time infusion, is currently undergoing a Phase I/IIa clinical trial in the United States. This major step emphasizes the company’s commitment to advancing gene therapies and providing new treatment options for patients with severe medical needs.
See more data about QURE stock on TipRanks’ Stock Analysis page.