Drugs / ACTUS-101
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT03533673 | Dec 2018 → Sep 2022 | glycogen storage disease due to acid maltase deficiency, late-onset | AskBio Inc | Completed | No outcome recorded |
Press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2019-01-22 | AskBio Inc | First Patient Dosed with Gene Therapy in Phase 1/2 Study of ACTUS-101 in Patients with Pompe Disease askbio.com ↗ |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ACTUS-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03533673 ↗ |
| Action | Restore | “enable expression of a functional copy of the GAA gene” NCT03533673 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Gene therapy with an adeno-associated virus serotype 8 (AAV8) vector” PMID 36805083 ↗ Feb 2023 |
| Route | Intravenous | “ACTUS-101 administered intravenously” NCT03533673 ↗ |
| Target | GAA | “ACTUS-101 is intended to enable expression of a functional copy of the GAA gene” NCT03533673 ↗ |