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 Alipogene 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 emparvovec Not Annotated Valoctocogene roxaparvovec An adeno-associated virus serotype 5 (AAV5)-based gene therapy containing a coagulation factor VIII complementary DNA used to treat severe hemophilia A. 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)). It is an adeno-associated virus (AAV) vector that... CERE-110 Investigated for use/treatment in alzheimer's disease and gene therapy. Eladocagene exuparvovec A recombinant AAV2 vector containing the human aromatic L-amino acid decarboxylase (AADC) gene indicated for the treatment of AADC deficiency. 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 Fidanacogene 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 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 Adlinacogene 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 civaparvovec Tefidsogene 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 civaparvovec Devafidugene 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 dezaparvovec An adeno-associated virus vector-based gene therapy used for the treatment of hemophilia B (congenital factor IX deficiency). 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 A gene therapy used to treat Duchenne Muscular Dystrophy in ambulatory children with DMD gene mutation. Cobnabexagene anvuparvovec Not Annotated Bomtabegagene bavoparvovec Not Annotated Verbrinacogene setparvovec Not Annotated Bevufenogene nofeparvovec Not Annotated Patidistrogene bexoparvovec Not Annotated Isaralgagene civaparvovec Isaralgagene 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 cosaparvovec Not Annotated Domofenogene zalfaparvovec Domofenogene zalfaparvovec, formerly known as BMN-307, is an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy developed by BioMarin Pharmaceutical. Ifezuntirgene inilparvovec Not Annotated Peboctocogene camaparvovec Not Annotated Aguracingene cadoparvovec Not Annotated Botaretigene sparoparvovec Not Annotated Laruparetigene zovaparvovec Not Annotated Lixmabegagene relduparvovec Lixmabegagene 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 soroparvovec Not Annotated Engabexagene cincesparvovec Not Annotated Zaftuclenegene piruparvovec Not Annotated NLX-P101 Investigated for use/treatment in parkinson's disease. Nadofaragene firadenovec A 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
Drug Target Type Voretigene neparvovec Retinoid isomerohydrolase target Valoctocogene roxaparvovec Coagulation factor VIII (F8) target