Drugs / AskBio009
AskBio009
also known as
AAV8.sc-TTR-FIXR338Lopt
Gene therapy (AAV / viral vector) targets F9 via restoration
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT01687608 | Feb 2013 → Jan 2030 expected | hemophilia B | Baxalta now part of Shire | Active not recruiting | No outcome recorded |
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AskBio009 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01687608 ↗ |
| Known as | AAV8.sc-TTR-FIXR338Lopt | “This phase 1/2, open-label dose-escalation study investigated BAX 335 (AskBio009, AAV8.sc-TTR-FIXR338Lopt), an adeno-associated virus serotype 8 (AAV8)-based FIX Padua gene...” PMID 33067633 ↗ Feb 2021 |
| Action | Restore | “designed to compensate for low FIX protein expression levels by expressing the FIX Padua variant” PMID 30003118 ↗ Jun 2018 |
| Modality | Gene therapy (AAV / viral vector) | “an adeno-associated virus serotype 8 (AAV8)-based FIX Padua gene therapy” PMID 33067633 ↗ Feb 2021 |
| Route | Intravenous | “Single dose IV injection” NCT01687608 ↗ |
| Target | F9 | “delivering a functional human F9 gene into liver cells” PMID 33067633 ↗ Feb 2021 |