uniQure

In today's investor conference call and webcast, uniQure discussed the updated interim data demonstrating slowing of disease progression in our ongoing #huntingtonsdisease Phase I/II clinical trials of #genetherapy AMT-130. The webcast recording is now available: https://lnkd.in/ecztjqY2

Today we announced positive interim data demonstrating slowing of disease progression in our ongoing Ph. I/II clinical trials of AMT-130 in #huntingtonsdisease. Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 24 months compared to an external control group. In addition, a statistically significant reduction of CSF NfL (a key biomarker of neurodegeneration) was observed in patients treated with AMT-130 compared to baseline. Based on data observed to date, AMT-130 remains generally well-tolerated, with a manageable safety profile at both doses. https://lnkd.in/eDcCyCmE

uniQure has entered into an agreement for the sale of our global manufacturing facility in Lexington, Massachusetts to Genezen, a leading contract development and manufacturing organization. “Today marks a significant step forward in achieving a key strategic goal for uniQure,” stated Matt Kapusta, chief executive officer of uniQure. “We have been focused on meaningfully reducing costs while maintaining our ability to develop and potentially commercialize our #genetherapy product candidates. This transaction achieves these objectives by unlocking significant cost savings while maintaining preferential access to world-class gene therapy manufacturing.” https://lnkd.in/eKnPqHVN

uniQure was proud to get out this weekend and support the Huntington Disease Society of America (HDSA)’s Team Hope Walk at Castle Island in Boston! #HDSA #genetherapy

uniQure sponsored and attended this week's European Network to Cure ALS (ENCALS) meeting in Sweden, where we presented posters on our SOD1 #ALS program (AMT-162) and preclinical C9orf72 program. Our team appreciated the opportunity to engage in the scientific and clinical sessions as well as connect with KOLs and peers. #genetherapy

We want to extend our deep gratitude and heartfelt thanks to Paula Soteropoulos as she steps down today from our Board of Directors after more than 10 years of dedicated service and commitment to uniQure. Thank you for everything, Paula! We wish you all the best in your future endeavors. 

We're happy to announce that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for AMT-130 for #huntingtonsdisease. AMT-130 becomes the first investigative therapy in HD ever to receive the designation, which was created to expedite the development and review processes of #genetherapy and other regenerative medicines. https://lnkd.in/e67ARSuS

Happy #PrideMonth! As a cornerstone of our company culture, we strive to create a diverse workplace where you can truly be yourself. We believe that when you bring your authentic self to work, you find greater meaning and fulfillment in what you do.

In recognition of HD Awareness Month, it was our privilege today to welcome HD community advocate Lauren Holder to speak to uniQure employees. Lauren is host of the podcast “Help4HD Live,” a member of the HD-CAB community advisory board, and a Huntington's Disease Youth Organization (HDYO) Ambassador. She shared with us her family's experience with #huntingtonsdisease and her work to support and give voice to a patient community in desperate need of treatment options.  

At uniQure, we remain steadfast in our commitment to corporate social responsibility. We perceive these initiatives not only as ethical imperatives but also instrumental in mitigating business risk, enhancing organizational effectiveness, and attracting, retaining and inspiring talent. We've updated our corporate responsibility report to highlight our continued efforts and commitment to important environmental, social and governance initiatives. Learn more here: https://lnkd.in/egp_3gfx