Boosting factor VIII production for people with hemophilia A
Developing hemophilia A therapeutics by targeting translational and posttranslational regulation of FVIII
This project tests small medicines that help certain faulty factor VIII genes produce more working protein for people with hemophilia A.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cleveland Clinic Lerner Com-Cwru NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project ID | NIH-11289337 on NIH RePORTER |
What this research studies
Researchers are looking for drugs that increase how much functional factor VIII protein is made, folded, and transported in cells with common hemophilia A mutations. They will use engineered cells and laboratory models carrying nonsense and missense FVIII mutations to screen and optimize small molecules that raise FVIII levels. The team will also explore ways to improve AAV gene therapy by enabling therapeutic FVIII expression at lower viral doses. Early work is done in the lab and could lead to later clinical testing if promising molecules are identified.
Who could benefit from this research
Good fit: Ideal candidates would be people with hemophilia A caused by nonsense or misfolding (missense) FVIII mutations who are interested in early-stage therapeutic options.
Not a fit: People with other bleeding disorders, hemophilia B, or complete deletions of the FVIII gene (no targetable protein) are unlikely to benefit from these approaches.
Why it matters
Potential benefit: If successful, this could let some people with hemophilia A raise their own factor VIII levels with lower-cost medicines or enable safer, lower-dose gene therapy, reducing bleeding and treatment burden.
How similar studies have performed: Related approaches like read-through drugs and small-molecule chaperones have had limited success in other genetic disorders and are still early or unproven for hemophilia A, so this is partly novel.
Where this research is happening
Cleveland, United States
- Cleveland Clinic Lerner Com-Cwru — Cleveland, United States (Active)
Researchers
- Principal investigator: Zhang, Bin — Cleveland Clinic Lerner Com-Cwru
- Study coordinator: Zhang, Bin
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.