Adstiladrin
nadofaragene firadenovec · IFN-alpha2b (adenovirus, intravesical)22 (derived from the dosing and observation schedule)
Imlygic: how long were the pivotal tox studies?
talimogene laherparepvec · GM-CSF (oncolytic HSV-1)
Longest pivotal study
12 (derived from the dosing and observation schedule)
“Exploratory and GLP-compliant repeat dose toxicity studies were conducted in Balb/c mice with up to 12 weekly doses.”
Pivotal GLP studies
GLP SC repeat-dose studies in BALB/c mice, up to 12 weekly doses; GLP biodistribution and mouse embryo-fetal studies
“Pivotal repeat-dose toxicology, biodistribution, and embryo-foetal development studies were performed in accordance with Good Laboratory Practice (GLP) regulations.”
Route in tox
Subcutaneous (pivotal); intratumoural, IV also used
“single or repeated doses of talimogene laherparepvec administered by SC, IV, or intratumoural injection were well tolerated in immunocompetent mice, rats, and dogs.”
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
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
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