Drug intelligence / Profile preview

GNR-038

Development stage
Phase 1
Lead developer
AO GENERIUM
Modality
Blood Coagulation Factors → Therapeutic Enzymes → Recombinant Proteins and Enzymes
Administration
Intravenous
01

Overview

GNR-038 is a recombinant C1 inhibitor (rhC1-inh), developed as a complete structural and functional analogue of the plasma C1 inhibitor. It was being developed by GENERIUM Pharmaceuticals for the treatment of hereditary angioedema, a rare genetic disorder characterized by recurrent episodes of severe swelling. The drug acts as a replacement therapy to compensate for deficient or dysfunctional C1 esterase inhibitor activity in patients with hereditary angioedema. Its mechanism involves modulation of the serpin family G member 1 (C1-INH), which plays a key role in controlling inflammation and vascular permeability. Development was discontinued after Phase I clinical trials[2][3][4][5].

02

Targets

MASP2 (Mannan-binding lectin-associated serine protease 2)MASP1 (MASP-1)C1S (Complement component 1s subcomponent serine protease)FXIIa (Coagulation Factor XIIa)KLK6 (Kallikrein-related peptidase 6)C1R (Complement component 1r subcomponent)

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