A new targeted drug to block harmful complement activity in blood disorders
Official title: Development of a first-in-class complement inhibitor for treating complement-mediated hematologic diseases
Funded at Cleveland Clinic Lerner Com-Cwru · NIH-11184452 · Data updated 2026-11-12
A novel nanobody drug designed to block the complement system to help people with PNH or autoimmune hemolytic anemia.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cleveland Clinic Lerner Com-Cwru NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | September 5, 2024 |
| Project end | July 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $563,500 |
| NIH project number | 5R61HL171747-02 |
| Project ID | NIH-11184452 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with paroxysmal nocturnal hemoglobinuria (PNH), autoimmune hemolytic anemia (AIHA), or other blood disorders driven by complement activation could be the main candidates for this approach.
✕Less likely to help
Patients whose anemia or blood problems are not caused by complement activation or who cannot receive biologic therapies may not benefit from this treatment.
What this research studies
Researchers are creating a new nanobody-based medicine that aims to stop the complement system from attacking red blood cells. In the first phase they will make and thoroughly characterize several candidate molecules in the lab. In the second phase they will test the lead candidate in preclinical models of PNH and autoimmune hemolytic anemia to measure how well it prevents hemolysis and how the drug spreads and clears in the body. If those results are promising, the drug could move toward clinical testing in patients.
Why it matters
Potential benefit: If successful, this treatment could reduce red blood cell destruction, improve anemia, and lower the risk of blood clots in people with complement-driven blood disorders.
How similar studies have gone: Existing complement-blocking drugs help some patients but have limitations, and nanobody-based inhibitors are a newer approach with encouraging early lab results but not yet tested in people.
Where this research is happening
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Cleveland Clinic Lerner Com-Cwru
Cleveland, United States Active
Researchers
- Principal investigator: Lin, Feng C — Cleveland Clinic Lerner Com-Cwru
- Study coordinator: Lin, Feng C
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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.