Adstiladrin
nadofaragene firadenovec · IFN-alpha2b (adenovirus, intravesical)22 (derived from the dosing and observation schedule)
Elevidys: how long were the pivotal tox studies?
delandistrogene moxeparvovec · Micro-dystrophin
Longest pivotal study
24 weeks
“A 6-month good laboratory practice (GLP) toxicology and biodistribution study (Report No. SR-19-050) evaluated single IV administration of 1.33 × 1014 and 4.01 × 1014 vg/kg ELEVIDYS”
Pivotal GLP studies
GLP single-dose IV mouse studies: 24-week (WT+mdx), 12-week (WT+mdx, Catalent lot), 13-week neonatal juvenile tox
“A 3-month GLP toxicology study (Report No. SR-20-015) evaluated single IV administration of ELEVIDYS (manufactured by Process B at Catalent) in neonatal C57BL/6J WT mice administered 1.33 × 1014 and 4.01 × 1014 vg/kg at post-natal day 1.”
Route in tox
Single intravenous injection (temporal vein in neonates)
“Dosing Regimen Single administration”
Same question, other gene therapies
Beqvez
fidanacogene elaparvovec · Factor IX104 weeks
Casgevy
exagamglogene autotemcel · BCL11A enhancer (CRISPR-Cas9, ex vivo HSC)20 weeks
Fayuvi
rebisufligene etisparvovec · SGSH (AAV9)26 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