Hemgenix (etranacogene dezaparvovec-drlb) - CSL Behring
Adstiladrin (nadofaragene firadenovec-vncg) - Ferring
Zolgensma (onasemnogene abeparvovec-xioi) - Novartis
Kebilidi (eladocagene exuparvovec-tneq) - PTC Therapeutics
Roctavian (valoctocogene roxaparvovec-rvox) - BioMarin
Elevidys (delandistrogene moxeparvovec-rokl) - Sarepta Therapeutics, Roche
Glybera (alipogene tiparvovec) - uniQure, Chiesi, Xenon
Luxturna (voretigene neparvovec-rzyl) - Novartis, Roche
https://pag.virtual-meeting.org/US/asgct2026/en-US/pag/presentation/684882
 
Ching-Ha Lai1,2
 
Mar 22, 2026
 
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