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nusinersen-SNA
- Development stage
- Preclinical
- Lead developer
- Biogen
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
- Administration
- Intrathecal
01
Overview
Nusinersen-SNA is an investigational antisense oligonucleotide (ASO) therapy developed by Exicure in collaboration with The Ohio State University Wexner Medical Center for the treatment of spinal muscular atrophy (SMA). It utilizes Exicure's proprietary Spherical Nucleic Acid (SNA) technology to formulate nusinersen, the active ingredient in Spinraza, into a dense, radial nanoparticle structure. This SNA format is designed to enhance cellular uptake and potency compared to traditional linear ASOs. Preclinical studies in SMA patient-derived cells and mouse models have shown that nusinersen-SNA significantly increases the production of stable SMN2 mRNA and functional survival motor neuron (SMN) protein, demonstrating up to a 45-fold increase in mRNA levels compared to a 2.5-fold increase for the linear format.
- Other names
- nusinersen SNASNA-nusinersen
02
Targets
ISS-N1 (SMN2 intronic splicing silencer N1)
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