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VHH-siBACE1 conjugate
- Development stage
- Preclinical
- Lead developer
- VECT-HORUS
- 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, Nanobodies (VHH) → Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
- Administration
- Intrathecal, Intravenous, Subcutaneous
01
Overview
VHH-siBACE1 conjugate is a preclinical-stage therapeutic conjugate developed by Vect-Horus in collaboration with the Institut of Neurophysiopathology (INP) for the treatment of Alzheimer's disease. The conjugate consists of a variable domain of heavy chain-only antibody (VHH) targeting transferrin receptor 1 (TfR1) conjugated via click chemistry to a chemically stabilized small interfering RNA (siRNA) targeting beta-secretase 1 (BACE1). By targeting TfR1, the VHH moiety facilitates receptor-mediated transcytosis across the blood-brain barrier (BBB) and cellular uptake into the central nervous system. Once inside the target cells, the siBACE1 payload mediates the knockdown of BACE1 mRNA, thereby reducing BACE1 expression and potentially mitigating amyloid pathology.
- Other names
- VHH-siBACE1VHH-siBACE-1VHH-siBACE 1
02
Targets
BACE1 (Beta-site APP-cleaving enzyme 1)BACE2 (Beta-site amyloid precursor protein cleaving enzyme 2)TFRC (Transferrin Receptor)
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