shortened complement factor H gene therapy

Shortened complement factor H gene therapy is an investigational **AAV-based gene therapy** being developed by **4D Molecular Therapeutics** for **age-related macular degeneration**. It delivers a **truncated form of complement factor H** to retinal tissues with the goal of restoring regulation of the **alternative complement pathway**, thereby reducing pathologic complement activation associated with retinal degeneration. The therapeutic concept is to increase local expression of functional shortened CFH after **intravitreal administration**, with biology centered on control of **complement component 3 mediated alternative pathway activity**. Preclinical work has also shown proof of concept for complement-mediated disease modulation in models such as **C3 glomerulonephritis**.

Other names

shortened complement factor H gene therapyshortened complement factor HsCFHtruncated complement factor H gene therapy