Drug intelligence / Profile preview

ALN-4915

Development stage
Phase 1
Lead developer
Alnylam 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
01

Overview

ALN-4915 is an investigational RNA interference (RNAi) therapeutic being developed by Alnylam Pharmaceuticals in collaboration with Regeneron. It consists of a small interfering RNA (siRNA) designed to target and silence the expression of Complement Component 3 (C3), a central protein in the complement cascade. The drug is specifically engineered for ocular delivery using Alnylam's proprietary IKAROS technology, which enables potent and durable gene silencing in the eye following intravitreal injection. ALN-4915 is primarily being evaluated for the treatment of Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD), where overactivation of the complement system is known to drive retinal cell death and vision loss.

02

Targets

C3

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