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nusinersen
- Development stage
- Approved
- Lead developer
- Biogen
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
Nusinersen is an antisense oligonucleotide designed to treat spinal muscular atrophy (SMA), a rare neuromuscular disorder caused by mutations in the SMN1 gene that result in deficiency of survival motor neuron (SMN) protein. Nusinersen works by binding to a specific sequence on the SMN2 pre-mRNA, modifying its splicing to promote inclusion of exon 7, thereby increasing production of full-length functional SMN protein. This compensates for the lack of SMN protein due to defective SMN1 and helps restore motor neuron function. Nusinersen is administered intrathecally and is approved for use in infants, children, and adults with SMA[1][2][3][4][5][6].
- Brand names
- Spinraza
- Other names
- nusinersen
02
Targets
ISS-N1 (SMN2 intronic splicing silencer N1)
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