Nice to see Uniqure have received the IND approval now to go ahead with their AMT-130 Huntington's disease trial.
Originally published on January 29, 2019 Dutch-American company uniQure has received approval from the US drug regulator the FDA, to begin the first ever gene therapy trial in Huntington’s Disease. Their plan is to use a virus, injected into the brain, to turn cells into a factory that makes a weapon to lower the harmful huntingtin protein.
I doubt Benitec will publish anything on it, but good to see ddRNAi and what was at least once Benitec IP going into the clinic - as in now, for a disease target where it has a good chance of making a therapeutic difference. We weren't totally mad being excited about BLT's once impressive IP portfolio.
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