Drug intelligence / Profile preview

complement factor H

Development stage
Preclinical
Lead developer
Disc Medicine
Modality
Recombinant Proteins and Enzymes
Administration
Intravitreal, Intravenous
01

Overview

Complement factor H is a **protein replacement biologic** comprising human factor H, a large soluble regulator of the alternative complement pathway. Therapeutically administered factor H is intended to restore deficient or dysfunctional complement regulation by binding **C3b**, acting as a cofactor for factor I-mediated cleavage of C3b, and accelerating decay of the alternative-pathway C3 convertase, thereby reducing pathologic complement activation. Development has included **GEM-103** from Gemini Therapeutics as a recombinant human factor H program for **dry age-related macular degeneration**, which reached phase 2 but failed to meet clinical endpoints and was terminated, and a **plasma-derived human complement factor H** program from LFB for **atypical hemolytic uremic syndrome associated with inherited complement abnormalities**, which received orphan designation. The name is non-specific because it can refer either to the endogenous protein itself or to more than one therapeutic product based on factor H.

Other names
factor Hhuman complement factor Hrecombinant human complement factor Hrecombinant human factor H
02

Targets

C3b (Complement Component 3b)CFI (Complement factor I)

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