Creating new treatments for hemophilia A by improving factor VIII production
Official title: Developing hemophilia A therapeutics by targeting translational and posttranslational regulation of FVIII
Funded at Cleveland Clinic Lerner Com-Cwru · NIH-11047704 · Data updated 2026-11-12
This study is looking at new ways to help people with hemophilia A by testing small drugs that can boost a key protein needed for blood clotting and exploring gene therapy options to provide longer-lasting solutions, making treatments easier and more affordable for patients.
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 | 2025 |
| Award (this year) | $466,266 |
| NIH project number | 5R01HL169427-02 |
| Project ID | NIH-11047704 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are males diagnosed with hemophilia A, particularly those with specific mutations affecting factor VIII production.
✕Less likely to help
Patients with hemophilia B or those who do not have mutations in the factor VIII gene may not benefit from this research.
What this research studies
This research focuses on developing innovative therapies for hemophilia A, a genetic disorder that affects blood clotting. The approach involves using small molecule drugs to enhance the production and function of the factor VIII protein, which is often deficient or dysfunctional in patients with this condition. By targeting specific mutations that cause hemophilia A, the research aims to improve patient access to effective treatments that are more affordable and easier to administer than current therapies. The study also explores the potential of gene therapy using adeno-associated viruses to provide long-term solutions for patients.
Why it matters
Potential benefit: If successful, this research could lead to more accessible and effective treatments for hemophilia A, improving the quality of life for patients.
How similar studies have gone: Previous research has shown promise in using small molecules and gene therapy for similar genetic disorders, indicating a potential for success in this approach.
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.