Ifezuntirgene inilparvovec is under clinical development by UniQure and currently in Phase II for Huntington Disease. According to GlobalData, Phase II drugs for Huntington Disease does not have sufficient historical data to build an indication benchmark PTSR for Phase II. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the Ifezuntirgene inilparvovec LoA Report. Buy the report here.
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Ifezuntirgene inilparvovec overview
Ifezuntirgene inilparvovec (AMT-130) is under development for the treatment of Huntington’s disease. The therapeutic candidate consists of Adeno-associated virus type 5 (AAV5) vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene. It is developed based on ddRNAi (miQURE) technology. DNA-directed RNAi (ddRNAi) involves the use of adeno-associated virus and thereby introduces DNA sequence directly to the nucleus of the cells. It is administered through intravenous route. The drug candidate is delivered through intra-striatal convection enhanced delivery (CED).
UniQure overview
UniQure develops and markets gene therapy products. The company develops adeno-associated virus (AAV) based gene therapies using its gene technology platform and offers disease-modifying treatments to patients with severe genetic diseases and other devastating diseases. UniQure’s lead product, AMT-060, is a gene therapy for the treatment of moderately-severe hemophilia B. Its preclinical products include AMT-130, an AAV5 vector for the treatment of Huntington’s disease and AMT-150 for Spinocerebellar Ataxia type 3. The company develops a gene platform to bring new disease modifying therapies to patients with genetic diseases. The company conducts drug discovery and preclinical research in collaboration with academic research institutions. UniQure is headquartered in Amsterdam, the Netherlands.
For a complete picture of Ifezuntirgene inilparvovec’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
