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CLN3-targeting spherical nucleic acid
- Development stage
- Preclinical
- Lead developer
- Exicure
- Modality
- Gene Therapies, 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, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
- Administration
- Intrathecal, Intraperitoneal, Intravenous, Intracerebroventricular, Topical
01
Overview
CLN3-targeting spherical nucleic acid is a preclinical nucleic acid therapeutic being developed by Exicure for CLN3 Batten disease, also known as juvenile neuronal ceroid lipofuscinosis. It uses Exicure's spherical nucleic acid platform, a nanoparticle-like three-dimensional architecture of densely oriented oligonucleotides designed to enter cells without conventional transfection agents. Based on the available description, the therapy is intended to target the **CLN3** gene and address the underlying monogenic cause of disease in patients with pathogenic CLN3 mutations, making it best characterized as an early-stage **gene-directed RNA therapy** for a rare lysosomal storage neurodegenerative disorder.
- Other names
- CLN3-targeting spherical nucleic acidCLN-3-targeting spherical nucleic acidCLN 3-targeting spherical nucleic acidCLN3-targeting SNACLN-3-targeting SNACLN 3-targeting SNA
02
Targets
CLN3 (Battenin)
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