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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.
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