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

Clinical Publications

• 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

• https://www.askbio.com/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
• Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease
• Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
• Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease
• Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease
• Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease