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TfR1-targeting MAPT siRNA conjugate
- Development stage
- Preclinical
- Lead developer
- Dyne Therapeutics
- 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
- Intravenous, Subcutaneous
01
Overview
TfR1-targeting MAPT siRNA conjugate is an investigational RNA interference (RNAi) therapeutic developed by Dyne Therapeutics using its proprietary FORCE™ platform. The drug consists of a small interfering RNA (siRNA) designed to silence all isoforms of the microtubule-associated protein tau (MAPT), conjugated to a delivery vehicle that targets the transferrin receptor 1 (TfR1). This targeting mechanism is intended to facilitate the transport of the siRNA across the blood-brain barrier (BBB) and into the central nervous system (CNS) following systemic (intravenous or subcutaneous) administration. By reducing tau protein expression, the conjugate aims to treat tauopathies, such as Alzheimer's disease, by preventing the pathological accumulation of tau aggregates. Preclinical studies in humanized mice and non-human primates have demonstrated robust and widespread MAPT RNA reduction throughout the brain, including deep brain regions like the caudate nucleus and putamen.
- Other names
- MAPT siRNA conjugateTfR1-mediated MAPT siRNATfR-1-mediated MAPT siRNATfR 1-mediated MAPT siRNA
02
Targets
MAPT (Microtubule-associated protein tau)TFRC (Transferrin Receptor)
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