{"id":"srp-9003","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Flu-like symptoms"},{"rate":null,"effect":"Transient liver enzyme elevation"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"SRP-9003 uses antisense technology to bind to and degrade mutant DMPK mRNA transcripts, thereby reducing the production of toxic DMPK protein that accumulates in muscle cells and causes progressive muscle weakness and dysfunction. By lowering DMPK protein levels, the drug aims to slow or halt disease progression in myotonic dystrophy type 1 (DM1), a genetic neuromuscular disorder.","oneSentence":"SRP-9003 is an antisense oligonucleotide that reduces production of DMPK protein by targeting mutant DMPK mRNA in myotonic dystrophy type 1.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:36:01.511Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Myotonic dystrophy type 1 (DM1)"}]},"trialDetails":[{"nctId":"NCT06246513","phase":"PHASE3","title":"A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4","status":"ACTIVE_NOT_RECRUITING","sponsor":"Sarepta Therapeutics, Inc.","startDate":"2024-01-15","conditions":"Limb-girdle Muscular Dystrophy","enrollment":17},{"nctId":"NCT05876780","phase":"PHASE1","title":"A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Sarepta Therapeutics, Inc.","startDate":"2022-12-19","conditions":"Limb Girdle Muscular Dystrophy","enrollment":6},{"nctId":"NCT03652259","phase":"PHASE1, PHASE2","title":"Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)","status":"TERMINATED","sponsor":"Sarepta Therapeutics, Inc.","startDate":"2018-10-27","conditions":"Limb-Girdle Muscular Dystrophy, Type 2E","enrollment":6}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec"],"phase":"phase_3","status":"active","brandName":"SRP-9003","genericName":"SRP-9003","companyName":"Sarepta Therapeutics, Inc.","companyId":"sarepta-therapeutics-inc","modality":"Biologic","firstApprovalDate":"","aiSummary":"SRP-9003 is an antisense oligonucleotide that reduces production of DMPK protein by targeting mutant DMPK mRNA in myotonic dystrophy type 1. Used for Myotonic dystrophy type 1 (DM1).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}