ISIS 333611

ISIS 333611 is a first-generation antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of familial amyotrophic lateral sclerosis (fALS) associated with mutations in the SOD1 gene. The drug is designed to selectively bind to SOD1 messenger RNA (mRNA), facilitating its degradation through an RNase H-dependent mechanism. By reducing the levels of SOD1 mRNA, the therapy aims to decrease the production of the superoxide dismutase 1 (SOD1) protein, which is believed to cause neurotoxicity through a gain-of-function mechanism in affected patients. ISIS 333611 was the first ASO to enter clinical trials for a neurodegenerative disease, administered via intrathecal injection to ensure delivery to the central nervous system. While the Phase 1 study established the safety and proof-of-concept for this modality in ALS, Ionis subsequently transitioned development efforts to more potent second-generation ASOs, such as tofersen.

Other names

ASO-SOD1ASO-SOD-1ASO-SOD 1