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AskBio009 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| AskBio009 Dose Escalation | EXPERIMENTAL | Single Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy |
| Name | Type | Description |
|---|---|---|
| AskBio009 | BIOLOGICAL | Single dose IV injection |
Inclusion Criteria: * Males age 18-75 years, inclusive * Established hemophilia B with ≥3 hemorrhages per year requiring treatment with exogenous FIX OR use of FIX prophylaxis because of history of frequent bleeding episodes * Plasma FIX activity ≤2% (\<1% for first cohort; then per protocol) * Neg...
AskBio009 is an investigational gene therapy being studied for the treatment of Hemophilia B in adults. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
AskBio009 targets Factor IX (FIX), the clotting protein that is deficient or defective in Hemophilia B. By delivering a functional copy of the Factor IX gene, the therapy aims to enable the body to produce the clotting factor.
AskBio009 is being developed by Takeda Pharmaceutical Company Limited (TSE: 4502, NYSE: TAK). The company is conducting a Phase 1 clinical trial to evaluate the therapy in adults with Hemophilia B.
AskBio009 is in Phase 1 clinical development. It is an investigational therapy and has not received FDA approval. The ongoing Phase 1 trial is active but not recruiting participants.
AskBio009 is being studied in a single Phase 1 clinical trial with the identifier NCT01687608. This open-label, uncontrolled trial is evaluating a single ascending dose of the gene therapy in 30 adult males with Hemophilia B in the United States.
AskBio009 is an adeno-associated virus serotype 8 (AAV8) Factor IX gene therapy. The clinical trial NCT01687608 is titled 'Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B.'