uniQure
@uniQure_NV
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uniQure is delivering on the promise of gene therapy - single treatments with potentially curative result for patients with severe genetic diseases.
Amsterdam, The Netherlands
Joined April 2012
Today we reported our financial results for the second quarter of 2025 and highlighted recent progress across our business.
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We were fortunate to attend and support @Help4HDI HIPE in Denver, CO. Where we spent time with the HD community and provided updates on our HD program.
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We were privileged to attend this past weekend’s #HDSAConvention to connect with and learn from the #huntingtonsdisease community. We return home inspired by their courage & resiliency and excited to continue our efforts to bring a transformative #genetherapy to families in need.
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The powerful candle-lighting ceremony at tonight’s HDSA National Awards Dinner & Gala was the emotional pinnacle of an inspiring evening, lighting the room with love and hope in memory of those lost and in honor of all those fighting against #huntingtonsdisease. #HDSAConvention
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"This is an exciting time in HD research, and there are some exciting updates with the AMT-130 program," said Dr. Sung. "For me, this falls under this umbrella of cautious optimism, which in the wake of failures that we've had in HD trials over many years, I will certainly take.".
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The two discussed the encouraging data in the AMT-130 clinical trial and the next steps for the program. They also expressed their deep appreciation for all the brave people who participate in clinical trials and make cutting-edge research like this possible.
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A full house at #HDSAConvention for today's Clinical Trial Showcase, which included a fireside chat between David Margolin, the program lead for our #huntingtonsdisease clinical program, and Dr. Victor Sung, director of the UAB HD clinic and a PI in the trial.
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Our #HDSAConvention booth is open! Come drop by this weekend to say hello, grab some snacks, and learn more about our AMT-130 #genetherapy program in #huntingtonsdisease.
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We're thrilled to be heading to Indy as a Gold Sponsor of this year's #HDSAConvention, where we'll have a fantastic cross-functional team on hand to attend the sessions, support the #huntingtonsdisease community, and share information about our AMT-130 #genetherapy program.
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CEO Matthew Kapusta talks about uniQure's mission to transform the lives of patients through the power of genetics, and how that mission drives who we are at uniQure. #genetherapy
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uniQure is proud to support the @End_the_Legacy conference! Join us to learn about the latest in genetic ALS and our commitment to treating it. #EndTheLegacy #GeneTherapy #uniQure
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Following recent Type B meetings, alignment with the FDA continues to support an Accelerated Approval pathway for AMT-130 in #Huntingtonsdisease. We've reached alignment with the FDA in several key areas, with a BLA submission expected in early 2026.
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We were happy to be a part of today's.@EpilepsyNE Boston Walk for #Epilepsy at historic Fenway Park. The Northeast's largest epilepsy event gave us and the NE epilepsy community a chance to walk through the beautiful neighborhood and spend time at the home of our beloved #RedSox!
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Today we'll present a clinical case study of the first participant dosed in the ongoing trial of #genetherapy AMT-260 in MTLE, showing no serious adverse events and 92% reduction in seizure frequency through the first five months of follow up. #epilepsy
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This #ALSAwarenessMonth, we reflect on the words of Ron Faretra, an #ALS advocate who spoke to our employees this year. His courage and determination reflect the strength of the ALS community and inspire us in our work to deliver a transformative #genetherapy for SOD1-ALS.
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We’ve had some bon temps in New Orleans this week at #ASGCT2025, where our team presented four posters and one oral presentation highlighting our leadership in #genetherapy research and capsid development.
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On this International #HDAwareness Day, we at uniQure are grateful for the HD advocates who have shared their powerful stories with our team over the years. Their experiences inspire us in our work to deliver a transformative treatment for the #HuntingtonsDisease community.
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Today we reported our financial results for the first quarter of 2025 and highlighted recent progress across our business, including initial safety data from the third cohort of the Phase I/II study of AMT-130 in #huntingtonsdisease.
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A beautiful spring day celebrating #HDawarenessmonth with @HDSA New England at today's Celebration of Hope brunch! As always, we are inspired by the indefatigable spirit of the #Huntingtonsdisease community and proud to lend our support to the wonderful work they do.
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uniQure is proud to support the ALS community at the ALS Roundtable in Washington, DC during ALS Awareness Month. We're excited to engage with leaders and advocates to advance research and solutions for ALS. Together, we can make a difference! 💙 #ALS #ALSAwarenessMonth
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