Askbio Inc
Clinical trials sponsored by Askbio Inc, explained in plain language.
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Gene therapy trial hopes to ease Parkinson's symptoms
Disease control Recruiting nowThis Phase 2 study tests whether a single dose of AAV2-GDNF gene therapy, delivered directly into the brain, can improve motor function in adults with moderate Parkinson's disease. About 127 participants will receive either the gene therapy or a sham surgery. The main goal is to …
Phase: PHASE2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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One-Time gene therapy could change pompe disease treatment
Disease control Recruiting nowThis study tests a single intravenous dose of a gene therapy called AB-1009 in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The main goal is to check safety and side effects, while also seeing if the tre…
Phase: PHASE1, PHASE2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Jun 27, 2026 12:38 UTC
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Gene therapy trial offers hope for rare muscle disease
Disease control Recruiting nowThis study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 10 meters in under 30 seconds.
Phase: PHASE1, PHASE2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC