Drug intelligence / Profile preview

ALN-4285

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: Subcutaneous

Overview

ALN-4285 is an investigational RNA interference (RNAi) therapeutic designed to treat complement-mediated diseases by targeting complement component C3. Developed by Alnylam Pharmaceuticals, it employs the company's GalNAc-conjugate technology for liver-specific delivery and the Enhanced Stabilization Chemistry Plus (ESC+) platform to improve potency and durability. By silencing the expression of C3, the central hub of the complement system, ALN-4285 aims to inhibit the classical, lectin, and alternative pathways, offering a potentially potent approach for diseases characterized by overactivation of the complement cascade. It is currently being evaluated in Phase 1 clinical trials for safety, pharmacokinetics, and pharmacodynamics.

Targets

Beyond the preview

Go deeper on ALN-4285.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on ALN-4285.

Explore the full profile