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
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 tiparvovecAlipogene tiparvovec is an adeno-associated viral vector of serotype 1-based gene therapy that delivers the Ser(447)X variant of the human lipoprotein lipase (LPL) gene. It is being investigated for the...
Timrepigene emparvovecNot Annotated
Valoctocogene roxaparvovecAn adeno-associated virus serotype 5 (AAV5)-based gene therapy containing a coagulation factor VIII complementary DNA used to treat severe hemophilia A.
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)). It is an adeno-associated virus (AAV) vector that...
CERE-110Investigated for use/treatment in alzheimer's disease and gene therapy.
Eladocagene exuparvovecA recombinant AAV2 vector containing the human aromatic L-amino acid decarboxylase (AADC) gene indicated for the treatment of AADC deficiency.
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 elaparvovecFidanacogene elaparvovec is an adeno-associated viral (AAV) vector-based gene therapy indicated for the treatment of adults (aged 18 years or older) with moderately severe to severe Hemophilia B (congenital Factor...
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 civaparvovecAdlinacogene civaparvovec is an investigational in vivo genome editing therapeutic product. It uses proprietary zinc finger nuclease (ZFN) genome editing to insert into the DNA of liver cells a copy...
Tefidsogene civaparvovecTefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing. It consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system...
Devafidugene civaparvovecDevafidugene civaparvovec is an investigational in vivo genome editing product. It uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme. Developed by Sangamo...
Etranacogene dezaparvovecAn adeno-associated virus vector-based gene therapy used for the treatment of hemophilia B (congenital factor IX deficiency).
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 moxeparvovecA gene therapy used to treat Duchenne Muscular Dystrophy in ambulatory children with DMD gene mutation.
Cobnabexagene anvuparvovecNot Annotated
Bomtabegagene bavoparvovecNot Annotated
Verbrinacogene setparvovecNot Annotated
Bevufenogene nofeparvovecNot Annotated
Patidistrogene bexoparvovecNot Annotated
Isaralgagene civaparvovecIsaralgagene civaparvovec, formerly known as ST-920, is liver-tropic rAAV 2/6 vector carrying the cDNA for human alpha-galactosidase A (α-Gal A) enzyme. It is developed by Sangamo Therapeutics and investigated in...
Sesiclenegene cosaparvovecNot Annotated
Domofenogene zalfaparvovecDomofenogene zalfaparvovec, formerly known as BMN-307, is an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy developed by BioMarin Pharmaceutical.
Ifezuntirgene inilparvovecNot Annotated
Peboctocogene camaparvovecNot Annotated
Aguracingene cadoparvovecNot Annotated
Botaretigene sparoparvovecNot Annotated
Laruparetigene zovaparvovecNot Annotated
Lixmabegagene relduparvovecLixmabegagene relduparvovec, formerly known as LYS-GM101, is a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (GBL1). It is under development for the treatment of GM1...
Ixoberogene soroparvovecNot Annotated
Engabexagene cincesparvovecNot Annotated
Zaftuclenegene piruparvovecNot Annotated
NLX-P101Investigated for use/treatment in parkinson's disease.
Nadofaragene firadenovecA non-replicating adenoviral vector-based gene therapy used to treat high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS).
Drugs & Drug Targets
DrugTargetType
Voretigene neparvovecRetinoid isomerohydrolasetarget
Valoctocogene roxaparvovecCoagulation factor VIII (F8)target