Drug intelligence / Profile preview

tau small interfering ribonucleic acid

Development stage
Unknown
Lead developer
Alnylam Pharmaceuticals
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intrathecal, Subcutaneous
01

Overview

Tau small interfering ribonucleic acid is a **tau-lowering RNA interference therapeutic concept** comprising small interfering RNA molecules designed to silence **MAPT mRNA**, thereby reducing production of tau protein in the central nervous system. This approach is being pursued for **Alzheimer's disease and other tauopathies**, based on the rationale that lowering intracellular tau may reduce tau aggregation, neurofibrillary tangle formation, and downstream neurodegeneration. The term as given is **not specific to one single named drug candidate** and can refer to multiple investigational programs, including intrathecal and subcutaneous CNS-directed MAPT siRNA candidates being developed by companies such as Eli Lilly, Arrowhead Pharmaceuticals, and Alnylam in preclinical or early clinical development.

Other names
tau small interfering ribonucleic acidtau siRNAMAPT siRNAmicrotubule-associated protein tau small interfering ribonucleic acid
02

Targets

MAPT (Microtubule-associated protein tau)

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