Clinical Trial: 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)

U.S., Dec. 16 -- ClinicalTrials.gov registry received information related to the study (NCT07282847) titled '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)' on Nov. 26.

Brief 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).

Study Start Date: Dec., 2025

Study Type: INTERVENTIONAL

Condition: Pompe Disease (Late-onset) Pompe Disease Late-Onset LOPD

Intervention: GENETIC: AB-1009 (GAA Gene)

A single intravenous infusion of AB-1009

Recruitment Status: NOT_YE...