Adeno-associated Viral Vector Therapies
- Name
- Adeno-associated Viral Vector Therapies
- Accession Number
- DBCAT005646
- Description
This category contains approved and investigational gene therapies utilizing adeno-associated virus (AAV) vectors. Gene therapies that use AAV vectors are denoted as such in standard gene therapy nomenclature by the infix -parvo- in the second part of its name.
- Drugs
Drug Drug Description Voretigene neparvovec An adeno-associated viral gene therapy used to treat biallelic RPE65 mutation associated retinal dystrophy. Onasemnogene abeparvovec A gene therapy used to treat neonatal and infant patients with spinal muscular atrophy caused by bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Alipogene tiparvovec Not Annotated Timrepigene emparvovec Not Annotated Valoctocogene roxaparvovec Valoctocogene roxaparvovec is under investigation in clinical trial NCT03392974 (Single-arm Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia a Patients at a Dose of 4E13 Vg/kg). Giroctocogene fitelparvovec Giroctocogene fitelparvovec is under investigation in clinical trial NCT04370054 (Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia a Adults). Olenasufligene relduparvovec Olenasufligene relduparvovec is under investigation in clinical trial NCT03612869 (Study of aavrh10-h.sgsh Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)). CERE-110 Investigated for use/treatment in alzheimer's disease and gene therapy. Eladocagene exuparvovec Not Annotated Resamirigene bilparvovec AT132 is under investigation in clinical trial NCT03199469 (Gene Transfer Clinical Study in X-linked Myotubular Myopathy). Lenadogene nolparvovec Not Annotated Lanacogene vosiparvovec Not Annotated Fidanacogene elaparvovec Not Annotated Rebisufligene etisparvovec Not Annotated Volrubigene ralaparvovec Volrubigene ralaparvovec is under investigation in clinical trial NCT03223194 (Gene Transfer Clinical Study in Crigler-najjar Syndrome). Inlezifigene civaparvovec Not Annotated Ranuzifigene civaparvovec Not Annotated Adlinacogene civaparvovec Not Annotated Tefidsogene civaparvovec Not Annotated Devafidugene civaparvovec Not Annotated Etranacogene dezaparvovec Not Annotated Dirloctocogene samoparvovec Not Annotated Rovoctocogene durparvovec Not Annotated Zildistrogene varoparvovec Not Annotated Ezaladcigene resoparvovec Not Annotated Avalotcagene ontaparvovec Not Annotated Cotoretigene toliparvovec Not Annotated Cevaretigene ritoparvovec Not Annotated Entacingene turiparvovec Not Annotated Fordadistrogene movaparvovec Not Annotated Pariglasgene brecaparvovec Not Annotated Delandistrogene moxeparvovec Not Annotated Cobnabexagene anvuparvovec Not Annotated Bomtabegagene bavoparvovec Not Annotated Verbrinacogene setparvovec Not Annotated Bevufenogene nofeparvovec Not Annotated Patidistrogene bexoparvovec Not Annotated Isaralgagene civaparvovec Not Annotated Sesiclenegene cosaparvovec Not Annotated Domofenogene zalfaparvovec Not Annotated Ifezuntirgene inilparvovec Not Annotated Peboctocogene camaparvovec Not Annotated Aguracingene cadoparvovec Not Annotated Botaretigene sparoparvovec Not Annotated Laruparetigene zovaparvovec Not Annotated Lixmabegagene relduparvovec Not Annotated Ixoberogene soroparvovec Not Annotated Engabexagene cincesparvovec Not Annotated Zaftuclenegene piruparvovec Not Annotated NLX-P101 Investigated for use/treatment in parkinson's disease. - Drugs & Drug Targets
Drug Target Type Voretigene neparvovec Retinoid isomerohydrolase target