A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

Phase 1/2 Recruiting 12 enrolled AskBio Inc Bayer · collab

Non-randomizedSequentialOpen-labelTreatment

Summary

This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).

Timeline

Start: 2026-04-15
Primary completion: 2028-09
Completion: 2032-09

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	AB-1009	Unknown	1e+13 vg/kg	Intravenous
Subject	AB-1009	Unknown	1.5e+13 vg/kg	Intravenous

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

Summary

Timeline

Drugs

Indications