Adstiladrin
nadofaragene firadenovec · IFN-alpha2b (adenovirus, intravesical)22 (derived from the dosing and observation schedule)
Otarmeni: how long were the pivotal tox studies?
lunsotogene parvec · OTOF (AAV, intracochlear)
Longest pivotal study
27 weeks
“A 7- and 27-Week Toxicity and Biodistribution Study Following a Single Intracochlear Injection of DB-OTO in Cynomolgus Monkeys”
Pivotal GLP studies
GLP single-dose: 2-month adult mouse (RWM), 3-month juvenile mouse (PSCC), 7/27-week cynomolgus (bilateral RWM)
“The 2-month Good Laboratory Practice (GLP) study in adult mice, and the 3-month GLP study in juvenile mice, at dose levels up to 1.3 x 1011 vg/ear showed no DB-OTO-related adverse findings, with a no observed adverse effect level (NOAEL) of 1.3 x 1011 vg/ear.”
Route in tox
Intracochlear via round window membrane; posterior semicircular canal in juvenile mice (worst-case systemic exposure)
“The intended clinical route of administration, IC injection via the round window membrane (RWM), was used in adult mice and monkeys, while a posterior semicircular canal (PSCC) injection was used in juvenile mice to assess a worst-case scenario for systemic exposure.”
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
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
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