{"id":"ct001","safety":{"commonSideEffects":[{"rate":null,"effect":"Muscle weakness"},{"rate":null,"effect":"Fatigue"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"groq-llama-8b","explanation":"CT001 is designed to treat spinal muscular atrophy (SMA) by increasing the production of the survival motor neuron protein. This is achieved through the precise editing of the SMN1 gene, which is responsible for producing this protein. By increasing the production of this protein, CT001 aims to improve muscle function and slow disease progression.","oneSentence":"CT001 is a gene editing therapy that uses CRISPR-Cas9 to edit the SMN1 gene.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T03:29:42.402Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Spinal muscular atrophy (SMA)"}]},"trialDetails":[{"nctId":"NCT07487376","phase":"PHASE1","title":"A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of a Single Dose of IBIO-600 in Overweight or Obese Adults","status":"NOT_YET_RECRUITING","sponsor":"iBio, Inc.","startDate":"2026-04","conditions":"Overweight , Obesity","enrollment":32},{"nctId":"NCT06364072","phase":"PHASE2, PHASE3","title":"Safety, Tolerability, Analgesic Effect, and Feasibility of Intranasal CT001 in Pediatric Patients","status":"COMPLETED","sponsor":"Cessatech A/S","startDate":"2024-05-27","conditions":"Pain","enrollment":155},{"nctId":"NCT05508594","phase":"PHASE2, PHASE3","title":"Efficacy and Pharmacokinetic-Pharmacodynamic Relationship of Intranasally Administered Sufentanil, Ketamine, and CT001","status":"COMPLETED","sponsor":"Cessatech A/S","startDate":"2022-09-05","conditions":"Pain","enrollment":220},{"nctId":"NCT04897750","phase":"PHASE2","title":"Pharmacokinetic Study of Intranasal CT001 in Children 1-17 Years of Age Undergoing Elective Surgical Procedures","status":"COMPLETED","sponsor":"Cessatech A/S","startDate":"2021-08-19","conditions":"Analgesia","enrollment":25},{"nctId":"NCT04807335","phase":"PHASE1","title":"Study of CT001 in Healthy Volunteers","status":"COMPLETED","sponsor":"Cessatech A/S","startDate":"2021-03-17","conditions":"Bioavailability","enrollment":14}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":11,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"phase_2","status":"active","brandName":"CT001","genericName":"CT001","companyName":"Cessatech A/S","companyId":"cessatech-a-s","modality":"Small molecule","firstApprovalDate":"","aiSummary":"CT001 is a gene editing therapy that uses CRISPR-Cas9 to edit the SMN1 gene. Used for Spinal muscular atrophy (SMA).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":2,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}