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ION286
- Development stage
- Unknown
- Lead developer
- Ionis Pharmaceuticals
- Modality
- 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, Cyclic Peptides → Modified Peptides → Peptides, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
ION286 (AZD4063) is an investigational therapeutic consisting of an oligonucleotide conjugated to a bicyclic peptide ligand developed by Ionis Pharmaceuticals in collaboration with Bicycle Therapeutics and AstraZeneca. The drug utilizes Bicycle's proprietary platform to target Transferrin Receptor 1 (TfR1), facilitating the transport of the oligonucleotide across the blood-brain barrier and into cardiac tissues via receptor-mediated transcytosis. This approach is designed to enable the treatment of cardiomyopathy and central nervous system (CNS) disorders by improving the delivery and uptake of nucleic acid medicines in traditionally difficult-to-reach organs. ION286 is currently in Phase 1 clinical development.
02
Targets
TFRC (Transferrin Receptor)
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