AB-1009 is an adeno-associated virus (AAV)-based gene therapy designed to address the underlying genetic cause of Pompe disease by enabling sustained production of the deficient enzyme acid alpha-glucosidase (GAA). The trial is assessing the safety, tolerability, and efficacy of the therapy in adults with LOPD across multiple sites in the United States.
LOPD is a rare, progressive genetic condition that causes worsening muscle weakness and respiratory complications. Although enzyme replacement therapies are available, there remains a significant unmet need for long-term treatment options.
The PROGRESS-GT LOPD study is expected to enroll approximately 12 participants in the United States. AB-1009 has received both Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration (FDA).
AskBio described this milestone as an important step forward in the development of potential new treatment approaches for people living with Pompe disease.
Source: Press release