Elixir Associates reposted this
UniQure's investigational gene therapy shown to slow Huntington's disease progression, impacting around 70,000 people in Europe and the US. Huntington's has no approved therapies or existing treatments that could delay the onset or slow the progression of this debilitating condition. UniQure has conducted two phase 1/2 clinical trials of its gene therapy candidate, AMT-130. The results after 24 months are promising. Among the 21 patients who participated, those receiving the high dose of AMT-130 demonstrated a statistically significant slowing in disease progression measured by the cUHDRS, a Huntington's disease rating scale. The data showed an 80% slowing of disease progression compared to a control group, with near-baseline stability in motor and cognitive function throughout the follow-up period. Additionally, there was a notable reduction in a key biomarker of neurodegeneration in patients treated with the high dose of AMT-130. Walid Abi-Saab, UniQure's chief medical officer, described this as a pioneering moment in Huntington's disease treatment, underscoring the potential for long-term clinical benefits and reduction in neurodegenerative markers. UniQure anticipates presenting this updated data to the FDA later this year to discuss potential expedited pathways for clinical development and accelerated approval. #Neurodegenrative #AAV #RareDiseases #GeneTherapy #HuntingtonsDisease #ElixirAssociates
