UniQure Submits Regulatory Applications for AMT-130 in Huntington's Disease
核心洞察
uniQure has filed a Biologics License Application with the FDA and a Marketing Authorization Application with the UK MHRA for ifezuntirgene inilparvovec, also known as AMT-130.
The Huntington's disease (搜索) gene therapy applications seek accelerated approval in the US and marketing authorization in the UK, supported by 3-year Phase I/II data.
Bristol Myers Squibb (搜索) reported positive topline Phase II results for GPRC5D (搜索)-directed CAR-T arlocabtagene autoleucel in quadruple-class exposed relapsed and refractory multiple myeloma (搜索).
uniQure has submitted regulatory applications for ifezuntirgene inilparvovec, also known as AMT-130, an investigational gene therapy for Huntington's disease (搜索). The company filed a Biologics License Application with the US FDA seeking accelerated approval and a Marketing Authorization Application with the UK's Medicines and Healthcare products Regulatory Agency. Both filings are supported by the previously announced 3-year data analysis from the Phase I/II clinical study of AMT-130. The therapy previously received Breakthrough Therapy and RMAT designations from the FDA.
Bristol Myers Squibb (搜索) announced positive topline results from the registrational Phase II QUINTESSENTIAL trial of arlocabtagene autoleucel, an autologous CAR-T therapy directed at GPRC5D (搜索), in adults with quadruple-class exposed relapsed and refractory multiple myeloma (搜索). The trial demonstrated a statistically significant and clinically meaningful overall response rate in patients who had received four or more prior lines of therapy, and met key secondary endpoints for response rates in this heavily pretreated population. The safety profile of arlo-cel was consistent with other CAR T cell and GPRC5D-targeting therapies for multiple myeloma.
EpilepsyGTx (搜索) and Viralgen (搜索) have formed a partnership, ahead of first-in-human clinical trials, to support production of EpilepsyGTx's EPY201, an investigational gene therapy programme for focal refractory epilepsy (搜索). EpilepsyGTx will use Viralgen's proprietary Aava manufacturing platform, which is designed to scale AAV programmes, for production of EPY201.