uniQure

uniQure has published a manuscript on our gene therapy AMT-191 in the journal "Molecular Therapy: Methods & Clinical Development." The publication details the preclinical work that demonstrated the potential of AMT-191 as a one-time treatment for Fabry disease and formed the foundation for our clinical trial that started this year. https://lnkd.in/eZua_iPr

Our investor conference call and webcast to discuss the announcement that we have aligned with the FDA on key elements of an Accelerated Approval pathway for our #genetherapy AMT-130 for #huntingtonsdisease will begin at 8:30 a.m. ET. https://lnkd.in/ecztjqY2.

Today uniQure announced that we have reached agreement with the FDA on key elements of an Accelerated Approval pathway for our #genetherapy AMT-130 for #huntingtonsdisease. The FDA has agreed that data from our ongoing Ph. I/II studies compared to a natural history external control may serve as the primary basis for a BLA for Accelerated Approval. https://lnkd.in/ekcYC7DV

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