Adstiladrin
nadofaragene firadenovec · IFN-alpha2b (adenovirus, intravesical)22 (derived from the dosing and observation schedule)
Fayuvi: how long were the pivotal tox studies?
rebisufligene etisparvovec · SGSH (AAV9)
Longest pivotal study
26 weeks
“No adverse vector-related findings were observed in the 6-month Good Laboratory Practice (GLP) safety and BD study”
Pivotal GLP studies
6-month GLP single IV study (5e12-5e13 vg/kg) and 3-month GLP single IV study (5e13-2e14 vg/kg) in WT C57BL/6 mice
“The definitive 3-month GLP safety and BD study investigated the effect of a single IV administration of 5.0x1013 to 2.0x1014 vg/kg FAYUVI in WT C57BL/6 mice.”
Route in tox
Intravenous
“single intravenous (IV) administration to wild type (WT) C57Bl/6 mice”
Same question, other gene therapies
Beqvez
fidanacogene elaparvovec · Factor IX104 weeks
Casgevy
exagamglogene autotemcel · BCL11A enhancer (CRISPR-Cas9, ex vivo HSC)20 weeks
Elevidys
delandistrogene moxeparvovec · Micro-dystrophin24 weeks
Genglycos
pariglasgene brecaparvovec · G6PC (AAV8)26 weeks
Glybera
alipogene tiparvovec · LPL (AAV1)26 weeks
Hemgenix
etranacogene dezaparvovec · Factor IX26 weeks
Imlygic
talimogene laherparepvec · GM-CSF (oncolytic HSV-1)12 (derived from the dosing and observation schedule)
Itvisma
onasemnogene abeparvovec (intrathecal) · SMN1 (AAV9, intrathecal)52 weeks
Kebilidi
eladocagene exuparvovec · AADC26 weeks
Kresladi
marnetegragene autotemcel · ITGB2 (LVV, ex vivo HSC)4 weeks
Lenmeldy
atidarsagene autotemcel · ARSA (LVV, ex vivo HSC)52 weeks
Luxturna
voretigene neparvovec · RPE6531 weeks
Lyfgenia
lovotibeglogene autotemcel · HBB T87Q (LVV, ex vivo HSC)26 weeks
Otarmeni
lunsotogene parvec · OTOF (AAV, intracochlear)27 weeks
Papzimeos
zopapogene imadenovec · HPV6/11 antigens (gorilla adenovirus)3 weeks
Roctavian
valoctocogene roxaparvovec · Factor VIII26 weeks
Skysona
elivaldogene autotemcel · ABCD1 (LVV, ex vivo HSC)13 (derived from the dosing and observation schedule)
Strimvelis
autologous CD34+ cells transduced with ADA (no INN) · ADA (gamma-retroviral, ex vivo HSC)16 weeks
Tudriqev
vusolimogene oderparepvec · GALV-GP + GM-CSF (oncolytic HSV-1)26 weeks
Vyjuvek
beremagene geperpavec · COL7A1 (HSV-1, topical)9 (derived from the dosing and observation schedule)
Waskyra
etuvetidigene autotemcel · WAS (LVV, ex vivo HSC)64 weeks
Zevaskyn
prademagene zamikeracel · COL7A1 (RV-modified keratinocyte sheets)8 weeks
Zolgensma
onasemnogene abeparvovec · SMN126 weeks
Zynteglo
betibeglogene autotemcel · HBB T87Q (LVV, ex vivo HSC)26 weeks