New ways to boost blood clotting for hemophilia A, hemophilia B, and factor X deficiency
Translating Mechanistic Insights into Intrinsic Xase Function
This work aims to find whether medicines that mimic factor VIII can help people with certain genetic forms of hemophilia B or factor X deficiency and to guide development of better clotting treatments.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11193800 on NIH RePORTER |
What this research studies
Researchers will study how the intrinsic Xase complex (the protein machinery that drives clotting) functions at the molecular level using biochemical and structural lab experiments. They will examine more than 40 hemophilia B (FIX) genetic variants and search for loss-of-function factor X variants that disrupt interactions with factor VIII. The team will test whether existing FVIII-mimetic drugs can restore clotting activity for select variants and use those results to design new therapeutic approaches. Work is led from Children's Hospital of Philadelphia and may involve collaboration with other centers for samples or future clinical translation.
Who could benefit from this research
Good fit: People with hemophilia B or factor X deficiency, especially those with specific loss-of-function FIX or FX gene variants that impair interactions with factor VIII, would be the most relevant candidates.
Not a fit: Patients whose bleeding is caused by unrelated mechanisms or who do not carry the specific responsive gene changes are unlikely to benefit directly from this work.
Why it matters
Potential benefit: If successful, this could expand effective treatment options by repurposing FVIII-mimetic drugs for some hemophilia B and factor X deficiency patients and inform new targeted therapies.
How similar studies have performed: Factor VIII‑mimetic drugs (for example, emicizumab) are already successful for many people with hemophilia A, and preliminary laboratory data suggest similar approaches could help some hemophilia B and FX variants, but clinical use in those groups remains largely untested.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Samelson-Jones, Ben J — Children's Hosp of Philadelphia
- Study coordinator: Samelson-Jones, Ben J
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.