Table 1.
Key viral vector-based gene therapy products in 2022.
| Drug | Development Stage | Mechanism of Action/Target Gene | Indication | Manufacture | Vector |
|---|---|---|---|---|---|
| ET140203 | Phase II Clinical Trial | Immuno-oncology therapy | Hepatocellular (liver) cancer (HCC) (including secondary metastases) | Eureka Therapeutics | Lentivirus |
| OTOF-GT | Preclinical | Otoferlin | Hearing loss general | Sensorion | Adeno-associated virus |
| AVR-RD-02 | Phase II Clinical Trial | Glucocerebrosidase beta | Gaucher’s disease | Avrobio | lentivirus |
| LX1004 | Phase II Clinical Trial | CLN2 | Neuronal ceroid lipofuscinosis (NCL) | Lexeo Therapeutics | Adeno-associated virus |
| SRP-9001 | Pre-registration | Micro-dystrophin | Duchenne muscular dystrophy (DMD) | Sarepta Therapeutics | Adeno-associated virus |
| Vyjuvek | Pre-registration | COL7A1 | Epidermolysis bullosa | Krystal Biotech | Herpes simplex virus |
| OCU400 | Phase II Clinical Trial | NR2E3 | Retinitis pigmentosa (RP) (ophthalmology) | Ocugen | Adeno-associated virus |
| Lumevoq | Pre-registration | ND4 | Leber’s hereditary optic neuropathy (LHON) | Genethon and GenSight Biologics | Adeno-associated virus |
| AVB-PGRN | Preclinical | Progranulin | Dementia, frontotemporal | AviadoBio | Adeno-associated virus |
| ADVM-022 | Phase II Clinical Trial | Aflibercept | wet age-related macular degeneration; diabetic retinopathy and other retinal conditions | Adverum Biotechnologies | Adeno-associated virus |
| ADVM-062 | Preclinical | L-opsin | Achromatopsia | Adverum Biotechnologies | Adeno-associated virus |
| 4D-125 | Phase II Clinical Trial | Retinitis pigmentosa GTPase regulator | Retinitis pigmentosa | 4D Molecular Therapeutics Roche | Adeno-associated virus |
| NR-082 | Phase III Clinical Trial | NADH dehydrogenase subunit 4 | Leber’s hereditary optic neuropathy | Wuhan Neurophth Biotechnology | Adeno-associated virus |
| ATA-100 | Phase II Clinical Trial | Fukutin related protein | Dystrophy, limb-girdle muscular, type 2I | Atamyo Therapeutics | Adeno-associated virus |
| SBT101 | Phase II Clinical Trial | ATP binding cassette subfamily D member 1 | Adrenoleukodystrophy | SwanBio Therapeutics | Adeno-associated virus |
| ASC-618 | Phase II Clinical Trial | B domain deleted liver-codon optimized factor VIII | Hemophilia A | ASC Therapeutics | Adeno-associated virus |