Drug intelligence / Profile preview

ALN-5288

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
Intrathecal
01

Overview

ALN-5288 is an investigational, intrathecally administered small interfering RNA (siRNA) developed by Alnylam Pharmaceuticals for the treatment of Alzheimer's disease and other tauopathies. It utilizes Alnylam's C16-conjugate technology, which is designed to enhance the delivery and uptake of the oligonucleotide into the tissues of the central nervous system (CNS). The drug works by targeting and silencing the messenger RNA (mRNA) that encodes the microtubule-associated protein tau (MAPT). By reducing the expression of tau protein, ALN-5288 aims to mitigate the formation and spread of neurofibrillary tangles, a core pathological feature of Alzheimer's disease associated with cognitive decline and neurodegeneration.

02

Targets

MAPT (Microtubule-associated protein tau)

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