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

Summary

Open-label, ascending dose trial of ACTUS-101 administered intravenously.

Timeline

Start

2018-12-17

Primary completion

2022-09-30

Completion

2026-03-12

Publications

• Smith EC, Hopkins S, Case LE, Xu M, Walters C, Dearmey S, Han SO, Spears TG, Chichester JA, Bossen EH, Hornik CP, Cohen JL, Bali D, Kishnani PS, Koeberl DD. Phase I study of liver depot gene therapy in late-onset Pompe disease. Mol Ther. 2023 Jul 5;31(7):1994-2004. doi: 10.1016/j.ymthe.2023.02.014. Epub 2023 Feb 18.

Drugs

Indications