Drug intelligence / Profile preview

ADP065

Development stage
Preclinical
Lead developer
Alector
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
01

Overview

ADP065 is a small interfering RNA (siRNA) therapeutic candidate developed by Alector. It is designed to target and silence the expression of the NLRP3 (NACHT, LRR and PYD domains-containing protein 3) gene. NLRP3 is a critical component of the NLRP3 inflammasome, a multi-protein complex that, upon activation, triggers the maturation and release of pro-inflammatory cytokines such as interleukin-1β (IL-1β) and interleukin-18 (IL-18). Chronic activation of the NLRP3 inflammasome is implicated in various neurodegenerative and systemic inflammatory diseases. By utilizing siRNA to reduce the levels of NLRP3 protein, ADP065 aims to modulate neuroinflammatory and systemic inflammatory pathways to treat conditions driven by excessive inflammasome activity. The program is currently in the early stages of development, appearing in Alector's pipeline for immuno-neurology and systemic inflammation.

02

Targets

NLRP3 (Nod-like receptor family pyrin domain-containing protein 3)

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