Astellas Gene Therapies
Clinical trials sponsored by Astellas Gene Therapies, explained in plain language.
A subsidiary of Astellas Pharma focused on gene therapy development.
Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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Gene therapy trial hopes to treat rare muscle disease
Disease control OngoingThis early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement ther…
Phase: PHASE1, PHASE2 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 13:07 UTC
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Gene therapy trial for rare muscle disease halted after severe side effects
Disease control OngoingThis trial tested a gene therapy called AT132 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that causes severe breathing problems. The therapy aimed to deliver a healthy copy of the MTM1 gene to improve muscle function. However, the study was st…
Phase: PHASE2, PHASE3 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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Gene therapy trial for rare heart condition halted before it began
Disease control TerminatedThis study was designed to test a new gene therapy called ASP2016 for heart problems caused by Friedreich Ataxia, a rare genetic disease. The therapy aimed to deliver a healthy copy of the faulty gene to help the heart. However, the study was withdrawn before any participants wer…
Phase: PHASE1 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Pompe disease study aims to clear path for gene therapy
Knowledge-focused OngoingThis study looks at people with late-onset Pompe disease to measure antibodies against a virus used in gene therapy and against the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine. About 119 teenagers and adults will give samples over 2 years…
Phase: NA • Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated Jul 19, 2026 00:00 UTC