Adeno-associated Viral Vector Therapies

Adeno-associated Viral Vector Therapies
Accession Number

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.

DrugDrug Description
Voretigene neparvovecAn adeno-associated viral gene therapy used to treat biallelic RPE65 mutation associated retinal dystrophy.
Onasemnogene abeparvovecA 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 tiparvovecNot Annotated
Timrepigene emparvovecNot Annotated
Valoctocogene roxaparvovecValoctocogene 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 fitelparvovecGiroctocogene 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 relduparvovecOlenasufligene relduparvovec is under investigation in clinical trial NCT03612869 (Study of aavrh10-h.sgsh Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)).
CERE-110Investigated for use/treatment in alzheimer's disease and gene therapy.
Eladocagene exuparvovecNot Annotated
Resamirigene bilparvovecAT132 is under investigation in clinical trial NCT03199469 (Gene Transfer Clinical Study in X-linked Myotubular Myopathy).
Lenadogene nolparvovecNot Annotated
Lanacogene vosiparvovecNot Annotated
Fidanacogene elaparvovecNot Annotated
Rebisufligene etisparvovecNot Annotated
Volrubigene ralaparvovecVolrubigene ralaparvovec is under investigation in clinical trial NCT03223194 (Gene Transfer Clinical Study in Crigler-najjar Syndrome).
Inlezifigene civaparvovecNot Annotated
Ranuzifigene civaparvovecNot Annotated
Adlinacogene civaparvovecNot Annotated
Tefidsogene civaparvovecNot Annotated
Devafidugene civaparvovecNot Annotated
Etranacogene dezaparvovecNot Annotated
Dirloctocogene samoparvovecNot Annotated
Rovoctocogene durparvovecNot Annotated
Zildistrogene varoparvovecNot Annotated
Ezaladcigene resoparvovecNot Annotated
Avalotcagene ontaparvovecNot Annotated
Cotoretigene toliparvovecNot Annotated
Cevaretigene ritoparvovecNot Annotated
Entacingene turiparvovecNot Annotated
Fordadistrogene movaparvovecNot Annotated
Pariglasgene brecaparvovecNot Annotated
Delandistrogene moxeparvovecNot Annotated
Cobnabexagene anvuparvovecNot Annotated
Bomtabegagene bavoparvovecNot Annotated
Verbrinacogene setparvovecNot Annotated
Bevufenogene nofeparvovecNot Annotated
Patidistrogene bexoparvovecNot Annotated
Isaralgagene civaparvovecNot Annotated
Sesiclenegene cosaparvovecNot Annotated
Domofenogene zalfaparvovecNot Annotated
Ifezuntirgene inilparvovecNot Annotated
Peboctocogene camaparvovecNot Annotated
Aguracingene cadoparvovecNot Annotated
Botaretigene sparoparvovecNot Annotated
Laruparetigene zovaparvovecNot Annotated
Lixmabegagene relduparvovecNot Annotated
Ixoberogene soroparvovecNot Annotated
Engabexagene cincesparvovecNot Annotated
Zaftuclenegene piruparvovecNot Annotated
NLX-P101Investigated for use/treatment in parkinson's disease.
Drugs & Drug Targets
Voretigene neparvovecRetinoid isomerohydrolasetarget