| Adstiladrinnadofaragene firadenovec · IFN-alpha2b (adenovirus, intravesical) |
|
|
|
|
|
|
→ |
| Beqvezfidanacogene elaparvovec · Factor IX |
|
|
|
|
|
|
→ |
| Casgevyexagamglogene autotemcel · BCL11A enhancer (CRISPR-Cas9, ex vivo HSC) |
|
|
|
|
|
|
→ |
| Elevidysdelandistrogene moxeparvovec · Micro-dystrophin |
|
|
|
|
|
|
→ |
| Fayuvirebisufligene etisparvovec · SGSH (AAV9) |
|
|
|
|
|
|
→ |
| Genglycospariglasgene brecaparvovec · G6PC (AAV8) |
|
|
|
|
|
|
→ |
| Glyberaalipogene tiparvovec · LPL (AAV1) |
|
|
|
|
|
|
→ |
| Hemgenixetranacogene dezaparvovec · Factor IX |
|
|
|
|
|
|
→ |
| Imlygictalimogene laherparepvec · GM-CSF (oncolytic HSV-1) |
|
|
|
|
|
|
→ |
| Itvismaonasemnogene abeparvovec (intrathecal) · SMN1 (AAV9, intrathecal) |
|
|
|
|
|
|
→ |
| Kebilidieladocagene exuparvovec · AADC |
|
|
|
|
|
|
→ |
| Kresladimarnetegragene autotemcel · ITGB2 (LVV, ex vivo HSC) |
|
|
|
|
|
|
→ |
| Lenmeldyatidarsagene autotemcel · ARSA (LVV, ex vivo HSC) |
|
|
|
|
|
|
→ |
| Luxturnavoretigene neparvovec · RPE65 |
|
|
|
|
|
|
→ |
| Lyfgenialovotibeglogene autotemcel · HBB T87Q (LVV, ex vivo HSC) |
|
|
|
|
|
|
→ |
| Otarmenilunsotogene parvec · OTOF (AAV, intracochlear) |
|
|
|
|
|
|
→ |
| Papzimeoszopapogene imadenovec · HPV6/11 antigens (gorilla adenovirus) |
|
|
|
|
|
|
→ |
| Roctavianvaloctocogene roxaparvovec · Factor VIII |
|
|
|
|
|
|
→ |
| Skysonaelivaldogene autotemcel · ABCD1 (LVV, ex vivo HSC) |
|
|
|
|
|
|
→ |
| Strimvelisautologous CD34+ cells transduced with ADA (no INN) · ADA (gamma-retroviral, ex vivo HSC) |
|
|
|
|
|
|
→ |
| Tudriqevvusolimogene oderparepvec · GALV-GP + GM-CSF (oncolytic HSV-1) |
|
|
|
|
|
|
→ |
| Vyjuvekberemagene geperpavec · COL7A1 (HSV-1, topical) |
|
|
|
|
|
|
→ |
| Waskyraetuvetidigene autotemcel · WAS (LVV, ex vivo HSC) |
|
|
|
|
|
|
→ |
| Zevaskynprademagene zamikeracel · COL7A1 (RV-modified keratinocyte sheets) |
|
|
|
|
|
|
→ |
| Zolgensmaonasemnogene abeparvovec · SMN1 |
|
|
|
|
|
|
→ |
| Zynteglobetibeglogene autotemcel · HBB T87Q (LVV, ex vivo HSC) |
|
|
|
|
|
|
→ |