{"id":"ro7234292-rg6042","safety":{"commonSideEffects":[{"rate":"null","effect":"Injection site reactions"},{"rate":"null","effect":"Nausea"},{"rate":"null","effect":"Vomiting"}]},"_chembl":{"chemblId":"CHEMBL4297581","moleculeType":"Oligonucleotide"},"_dailymed":null,"mechanism":{"_ai_source":"groq-llama-8b","explanation":"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.","oneSentence":"RO7234292 is a small molecule antisense oligonucleotide that targets the SMN2 gene to increase production of the survival motor neuron protein.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T01:32:33.562Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Spinal muscular atrophy"}]},"trialDetails":[{"nctId":"NCT05686551","phase":"PHASE2","title":"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","status":"ACTIVE_NOT_RECRUITING","sponsor":"Hoffmann-La Roche","startDate":"2023-02-03","conditions":"Huntington Disease","enrollment":301},{"nctId":"NCT04000594","phase":"PHASE1","title":"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","status":"COMPLETED","sponsor":"Hoffmann-La Roche","startDate":"2019-09-02","conditions":"Huntingtons Disease","enrollment":12},{"nctId":"NCT03761849","phase":"PHASE3","title":"A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease","status":"COMPLETED","sponsor":"Hoffmann-La Roche","startDate":"2019-01-23","conditions":"Huntingtons Disease","enrollment":899},{"nctId":"NCT03842969","phase":"PHASE3","title":"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","status":"COMPLETED","sponsor":"Hoffmann-La Roche","startDate":"2019-04-23","conditions":"Huntington Disease","enrollment":236},{"nctId":"NCT03342053","phase":"PHASE2","title":"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)","status":"COMPLETED","sponsor":"Hoffmann-La Roche","startDate":"2018-01-02","conditions":"Huntington's Disease","enrollment":46}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":1,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Tominersen"],"phase":"phase_3","status":"active","brandName":"RO7234292 (RG6042)","genericName":"RO7234292 (RG6042)","companyName":"Hoffmann-La Roche","companyId":"roche","modality":"Small molecule","firstApprovalDate":"","aiSummary":"RO7234292 (RG6042) is an antisense inhibitor that targets Huntingtin mRNA. It is being studied as a potential treatment for manifest Huntington's Disease through intrathecal administration in a clinical trial.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}