Zocaglusagene nuzaparvovec is under clinical development by Astellas Gene Therapies and currently in Phase II for Pompe Disease. According to GlobalData, Phase II drugs for Pompe Disease have a 63% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Zocaglusagene nuzaparvovec’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Zocaglusagene nuzaparvovec overview

Zocaglusagene nuzaparvovec (AT-845) is under development for the treatment of Pompe disease. The therapeutic candidate comprises of NAV adeno-associated virus 8 (AAV8) expressing acid alpha-glucosidase (GAA) gene. It is administered through intravenous route of administration.

Astellas Gene Therapies overview

Astellas Gene Therapies (Astellas Gene) is a biotechnology company that focuses on the development and commercialization of gene therapy products for rare diseases. The company’s major pipeline products include AT132, AT845, ASP2016, AT466 and KT430. It’s products are used for the treatment of X-linked myotubular myopathy, pompe disease, muscular dystrophy in children and neuromuscular disease, severe respiratory insufficiency, weakness and hypotonia. The company is currently exploring three gene therapy modalities such as gene replacement, exon skipping gene therapy, and vectorized RNA knockdown and will also advance additional Astellas gene therapy programs toward clinical investigation. Astellas Gene is headquartered in San Francisco, California, the US.

For a complete picture of Zocaglusagene nuzaparvovec’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 20 May 2024

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

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.