uniQure

We have successfully dosed the first patient in our clinical trial of AMT-260 in temporal lobe #epilepsy, representing an important milestone for uniQure and our third clinical trial initiation over the past six months. Nearly one-third of people experiencing focal onset seizures do not respond to currently available treatments and are left with limited therapeutic options – AMT-260 has the potential to be a transformative treatment option for these patients. https://lnkd.in/etfxiHbV

This week at #HSG2024, the uniQure team connected with academia, industry & clinicians focused on the fight against #huntingtonsdisease. We presented our AMT-130 #genetherapy program, met with our investigators and other HCPs, and attended engaging and informative sessions and discussions.

Happy Diwali from uniQure! We celebrated the festival of lights at our Lexington office with a delicious Indian lunch and beautiful henna designs from a talented artist. Wishing everyone a joyous and prosperous Diwali! #HappyDiwali2024

Today we reported our financial results for Q3 and highlighted recent progress across our business, having made significant strides during the third quarter both clinically and operationally. https://lnkd.in/eRdTT_jN

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