Last reviewed 2026-04-23 · How we verify

RO7234292 (RG6042)

RO7234292 is a small molecule antisense oligonucleotide that targets the SMN2 gene to increase production of the survival motor neuron protein.

RO7234292 is a small molecule antisense oligonucleotide that targets the SMN2 gene to increase production of the survival motor neuron protein. Used for Spinal muscular atrophy.

At a glance

Mechanism of action

This approach aims to treat spinal muscular atrophy by increasing the levels of the survival motor neuron protein, which is essential for motor neuron function and survival. By targeting the SMN2 gene, RO7234292 can potentially provide a long-term therapeutic effect.

Approved indications

• Spinal muscular atrophy

Common side effects

• Injection site reactions
• Nausea
• Vomiting

Key clinical trials

• GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease (PHASE2)
• A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease (PHASE1)
• A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease (PHASE3)
• An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies (PHASE3)
• A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139) (PHASE2)

Primary sources

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