uniQure

We're pleased to announce that we have a manuscript published in Molecular Therapy: Methods & Clinical Development that highlights our investigational AAV9-based #genetherapy candidate AMT-260 targeting GRIK2 in animal models and that details our approach to targeting mesial temporal lobe #epilepsy. https://lnkd.in/eU_rSJgd

Wrapping up a fantastic and productive week at the European Society of Gene and Cell Therapy Congress, where we presented seven posters highlighting our #genetherapy innovation and leadership. #ESGCT2024

We are pleased to announce the first patient dosing in the Phase I/II clinical trial of AMT-162, our investigational #genetherapy for the treatment of SOD1 #ALS, a debilitating, degenerative and fatal disease. https://lnkd.in/eXCUexYf

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure’s investigational #genetherapy for the treatment of #Fabry disease, a rare, inherited genetic disease. In August 2024, we announced the dosing of the first patient in our U.S. trial of AMT-191. https://lnkd.in/eaYWbWh6

There's a lot of excitement around uniQure's AMT-130 #genetherapy program at #EHDN2024! Today Prof. Ed Wild presented the latest AMT-130 data to this important meeting of the European #Huntingtonsdisease community.

As we prepare to dose the first patients in the clinical trial for AMT-162 for SOD1-related #ALS, we're pleased to have the program featured on the blog for the ALS Therapy Development Institute, the world's largest drug discovery lab focused solely on ending ALS. This educational piece explains our #genetherapy approach to reducing the toxic SOD1 proteins associated with ALS, hopefully leading to the halting or slowing of disease progression. It also explains the design of the trial and points to more information of patients who might be interested in learning more about this potential treatment option. https://lnkd.in/geycQf64

Today we announced that the first patient has been dosed in our Phase I/IIa clinical trial of #genetherapy AMT-191 for the treatment of #Fabry disease, a rare, inherited genetic disease. This achievement marks an exciting period for uniQure as we advance additional programs into clinical trials this year. https://lnkd.in/gmUb4Duj

After a comprehensive review, today uniQure announced an organizational restructuring intended to streamline operations, lower recurring cash burn by $75 million per year, and ensure we are optimally positioned for the future. https://lnkd.in/esXATAX3

Today we announced the closing of the sale of our global manufacturing facility in Lexington to Genezen, an important step toward achieving the company's goal of significantly reducing expenses and streamlining operations. https://lnkd.in/ezxatm-T

We're happy to join The ALS Association for its Nexus Conference in Dallas, where we'll have the opportunity to connect with the #ALS community and share more about our SOD1 #genetherapy clinical trial, which will be enrolling patients soon. https://lnkd.in/eNHFDhC8