Boosting factor VIII production for people with hemophilia A
Official title: Developing hemophilia A therapeutics by targeting translational and posttranslational regulation of FVIII
Funded at Cleveland Clinic Lerner Com-Cwru · NIH-11289337 · Data updated 2026-11-12
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 start | March 15, 2024 |
| Project end | January 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $492,171 |
| NIH project number | 5R01HL169427-03 |
| Project ID | NIH-11289337 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be people with hemophilia A caused by nonsense or misfolding (missense) FVIII mutations who are interested in early-stage therapeutic options.
✕Less likely to help
People with other bleeding disorders, hemophilia B, or complete deletions of the FVIII gene (no targetable protein) are unlikely to benefit from these approaches.
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.
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 gone: 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 Clinic Lerner Com-Cwru
Cleveland, United States Active
Researchers
- Principal investigator: Zhang, Bin — Cleveland Clinic Lerner Com-Cwru
- Study coordinator: Zhang, Bin
Related research
Other NIH-funded projects on the same topics.
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cGMP production of FVIII-making placental cells to help people with Hemophilia A
A new, patient-friendly therapy aims to make FVIII-producing placental cells in the lab to help adults with Hemophilia A, potentially offering a longer-lasting and more affordable treatment.
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A safer, longer-lasting gene delivery approach for Hemophilia A using improved nonviral vectors
Researchers are testing a new type of nonviral gene delivery that could provide longer expression of a therapeutic gene for adults with Hemophilia A.
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Hemophilia A: studying why some people develop inhibitors to FVIII across families
This NIH grant funds a multi-year program to follow families affected by severe hemophilia A to understand why some individuals develop antibodies against factor VIII.
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Intraosseous delivery of gene therapy vectors for hemophilia A
A gene therapy approach using vectors delivered into bone to help people with hemophilia A reduce or eliminate the need for frequent FVIII treatments.
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Targeting FVIII production to eye-opening blood vessels in hemophilia A
Aims to improve treatment for people with hemophilia A by directing how the body makes clotting factor VIII, using a targeted gene-delivery approach.
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Platelet-targeted FVIII Gene Therapy for Hemophilia A
A gene therapy approach aims to make platelets carry FVIII to help people with hemophilia A, even if they have inhibitors against FVIII.
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.