AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Clinical Publications

• (Abstract #1211) Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Initial 104-Week Experience - ASGCT 2022
• Screening data from 19 patients with late-onset Pompe disease for a phase I clinical trial of AAV8 vector-mediated gene therapy
• Phase I study of liver depot gene therapy in late-onset Pompe disease

News and Press Releases

• First Patient Dosed with Gene Therapy in Phase 1/2 Study of ACTUS-101 in Patients with Pompe Disease

Preclinical Publications

• Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease
• Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease
• Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
• Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease
• Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease
• Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease