{"id":"bmn-110","safety":{"commonSideEffects":[{"rate":null,"effect":"Immune response to AAV vector"},{"rate":null,"effect":"Liver enzyme elevation"},{"rate":null,"effect":"Thrombocytopenia"}]},"_chembl":{"chemblId":"CHEMBL3663415","moleculeType":"Small molecule","molecularWeight":"407.45"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Tay-Sachs disease is caused by deficiency of hexosaminidase A (Hex-A), leading to accumulation of GM2 ganglioside in neurons and progressive neurological deterioration. BMN 110 uses an adeno-associated virus (AAV) vector to deliver a functional HEXA gene directly to the central nervous system, enabling production of functional Hex-A enzyme and reducing pathogenic substrate accumulation. This approach aims to halt or slow neurological progression in affected patients.","oneSentence":"BMN 110 is a gene therapy that delivers a functional copy of the HEXA gene to restore hexosaminidase A enzyme activity in patients with Tay-Sachs disease.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:04:30.881Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Tay-Sachs disease (late-infantile or juvenile onset)"}]},"trialDetails":[{"nctId":"NCT02294877","phase":"","title":"A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2014-09","conditions":"Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA","enrollment":418},{"nctId":"NCT01415427","phase":"PHASE3","title":"Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2011-07","conditions":"Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA","enrollment":173},{"nctId":"NCT01966029","phase":"PHASE3","title":"BMN 110 Phase 3B in Australian Patients","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2013-07","conditions":"Mucopolysaccharidosis IVA (Morquio A Syndrome)","enrollment":13},{"nctId":"NCT01515956","phase":"PHASE2","title":"Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2011-10","conditions":"Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA","enrollment":15},{"nctId":"NCT01609062","phase":"PHASE2","title":"Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome","status":"TERMINATED","sponsor":"BioMarin Pharmaceutical","startDate":"2012-04","conditions":"Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA","enrollment":25},{"nctId":"NCT01697319","phase":"PHASE2","title":"Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation","status":"TERMINATED","sponsor":"BioMarin Pharmaceutical","startDate":"2012-08","conditions":"Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA","enrollment":16},{"nctId":"NCT01242111","phase":"PHASE1, PHASE2","title":"A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)","status":"TERMINATED","sponsor":"BioMarin Pharmaceutical","startDate":"2010-11","conditions":"MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome","enrollment":20},{"nctId":"NCT01275066","phase":"PHASE3","title":"A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2011-02","conditions":"MPS IV A","enrollment":177},{"nctId":"NCT00884949","phase":"PHASE1, PHASE2","title":"A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA","status":"COMPLETED","sponsor":"BioMarin Pharmaceutical","startDate":"2009-04","conditions":"MPS IV A","enrollment":20},{"nctId":"NCT01858103","phase":"","title":"BMN 110 US Expanded Access Program","status":"APPROVED_FOR_MARKETING","sponsor":"BioMarin Pharmaceutical","startDate":"","conditions":"Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA","enrollment":""}],"_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":["recombinant human N-acetylgalactosamine-6-sulfatase","N-acetylgalactosamine-6-sulfatase","N-acetylgalactosamine-6-sulfate","sulfatase","galactose-6-sulfatase"],"phase":"phase_3","status":"active","brandName":"BMN 110","genericName":"BMN 110","companyName":"BioMarin Pharmaceutical","companyId":"biomarin-pharmaceutical","modality":"Small molecule","firstApprovalDate":"","aiSummary":"BMN 110 is a gene therapy that delivers a functional copy of the HEXA gene to restore hexosaminidase A enzyme activity in patients with Tay-Sachs disease. Used for Tay-Sachs disease (late-infantile or juvenile onset).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}