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tominersen
- Development stage
- Phase 3
- Lead developer
- Roche
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
Tominersen is an experimental antisense oligonucleotide (ASO) therapy developed to treat Huntington's disease. It works by binding to the messenger RNA (mRNA) of the huntingtin (HTT) gene, which contains a mutation in people with Huntington's disease. By binding to HTT mRNA, tominersen marks it for destruction and reduces production of both normal and mutant huntingtin protein. This reduction aims to slow or prevent neurodegeneration associated with toxic clumps of mutant huntingtin protein in the brain. Tominersen is administered via intrathecal injection directly into cerebrospinal fluid because ASOs do not naturally cross into the brain or spinal cord from blood circulation[1][2][4][5].
- Other names
- tominersenRO7234292RO-7234292RO 7234292RG6042RG-6042RG 6042IONIS HTTRx
02
Targets
HTT mRNA (HTT / Huntingtin gene)
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