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GenSciP165

Development stage: Preclinical
Lead developer: Changchun GeneScience Pharmaceutical
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 Conjugates → Antibody-Based Therapeutics
Administration: Intravenous

Overview

GenSciP165 is an antibody-oligonucleotide conjugate (AOC) consisting of an antibody targeting the transferrin receptor (TfR) conjugated to a small interfering RNA (siRNA) targeting amyloid precursor protein (APP). Developed by GeneScience Pharmaceuticals (GenSci) using its blood-brain barrier delivery TransNeuro™ Platform, GenSciP165 is designed to cross the blood-brain barrier via TfR-mediated transcytosis and deliver the APP-targeting siRNA payload to the central nervous system. It is currently in preclinical development (preclinical candidate stage) for the treatment of Alzheimer's disease.

Targets

Aβ (Amyloid-beta peptides and aggregates)TFRC (Transferrin Receptor)

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