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APL-3007

Development stage: Phase 2
Lead developer: Apellis Pharmaceuticals
Modality: Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravitreal

Overview

APL-3007 is a small interfering RNA (siRNA) therapeutic developed by Apellis Pharmaceuticals. It acts as an inhibitor of complement component C3 expression via RNA interference, aiming to reduce the activation of the complement system implicated in various autoimmune and inflammatory diseases. The drug is being investigated primarily for the treatment of geographic atrophy, a leading cause of blindness associated with age-related macular degeneration. As of March 2025, APL-3007 is in Phase I clinical trials for this indication.

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