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ulefnersen
- Development stage
- Phase 3
- Lead developer
- Ionis Pharmaceuticals
- 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
- Administration
- Intrathecal
01
Overview
Ulefnersen is a next-generation antisense oligonucleotide (ASO) designed to treat amyotrophic lateral sclerosis (ALS) caused by mutations in the fused in sarcoma (FUS) gene. It works by silencing the FUS gene and reducing production of both toxic mutant and normal FUS proteins through RNA interference mechanisms. This targeted approach aims to prevent or delay motor neuron degeneration associated with this aggressive form of ALS. Ulefnersen was originally developed for compassionate use in a single patient but has since advanced into global phase 3 clinical trials after showing promising results, including slowing disease progression and even functional recovery in some patients. The drug is administered intrathecally every 12 weeks[2][5][6][9].
- Brand names
- jacifusenulefnersen sodium
- Other names
- jacifusenulefnersen sodium
02
Targets
FUS (Fused in sarcoma)
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