Drug intelligence / Profile preview

TXP1-oligoMAPT

Development stage
Preclinical
Lead developer
Ossianix
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, Antibody-Based Therapeutics
Administration
Intravenous
01

Overview

TXP1-oligoMAPT is a preclinical-stage brain shuttle-enabled small interfering RNA (siRNA) conjugate developed by Ossianix for the treatment of Alzheimer's disease and other tauopathies. The therapeutic consists of TXP1, a novel brain shuttle based on a single-domain Variable New Antigen Receptor (VNAR) anti-transferrin receptor 1 (TfR1) antibody derived from shark antibodies, site-specifically conjugated to an siRNA targeting microtubule-associated protein tau (MAPT). By targeting TfR1, TXP1 facilitates receptor-mediated transcytosis across the blood-brain barrier (BBB) to deliver the siRNA payload into the central nervous system, where it silences MAPT expression to reduce tau protein levels and mitigate tau pathology.

02

Targets

RISC (RNA-induced silencing complex)MAPT (Microtubule-associated protein tau)TFRC (Transferrin Receptor)

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