# Development of a first-in-class complement inhibitor for treating complement-mediated hematologic diseases

> **NIH NIH R61** · CLEVELAND CLINIC LERNER COM-CWRU · 2024 · $563,500

## Abstract

ABSTRACT
 Excessive complement activation causes many diseases especially hematologic diseases such as
paroxysmal nocturnal hemoglobinuria (PNH) and autoimmune hemolytic anemia (AIHA), in which patient
erythrocytes are attacked by activated complement, causing extravascular and intravascular hemolysis, thus
anemia, thrombosis, and other life-threatening complications. Several complement inhibitors have been
approved by the FDA for treating these disorders with limited success, thus there is still a clinical demand for
better complement-targeted therapeutics. In pilot studies, we have developed a novel nanobody-based
complement inhibitor and demonstrated its potential in treating complement-mediated hematologic diseases. In
this project, we will develop additional novel complement inhibitors and thoroughly characterize them (R61
Phase), then examine the lead candidate in preclinical models of PNH and AIHA for its treatment efficacies
and pharmacokinetics profiles (R33 Phase). This project, if successful, will identify a promising first-in-class
complement inhibitor for further clinical development into a much-needed new drug for patients with
complement-mediated diseases such as PNH and AIHA.

## Key facts

- **NIH application ID:** 10990215
- **Project number:** 1R61HL171747-01A1
- **Recipient organization:** CLEVELAND CLINIC LERNER COM-CWRU
- **Principal Investigator:** FENG C LIN
- **Activity code:** R61 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $563,500
- **Award type:** 1
- **Project period:** 2024-09-05 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10990215

## Citation

> US National Institutes of Health, RePORTER application 10990215, Development of a first-in-class complement inhibitor for treating complement-mediated hematologic diseases (1R61HL171747-01A1). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10990215. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*